Trial Outcomes & Findings for A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants (NCT NCT02908685)

Last Updated: 2024-04-24

Results Overview

The Internal Monitoring Committee (IMC) was responsible for selecting the dose for Part 2 of the study (pivotal dose). An external Independent Data Monitoring Committee (iDMC) reviewed data from Part 1 and confirmed the dose-selection decision of the IMC. The dose for Part 2 selected by the IMC was a dose that: 1.Was judged to be safe and well-tolerated, based on all available safety data from Part 1 and as confirmed by the iDMC; 2. Resulted in an exposure at steady-state below the exposure cap (mean value) of AUC0-24h,ss 2000 ng\*h/mL (adjusted for free-fraction, if required); 3. Resulted in an SMN protein increase that was expected to be clinically relevant.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

231 participants

Primary outcome timeframe

Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017)

Results posted on

2024-04-24

Participant Flow

Study Part 1 was conducted at 5 investigational sites across 4 countries, and Part 2 was conducted at 42 investigational sites across 14 countries. Screening in both Part 1 and 2 was up to 30 days prior to first dose.

In Part 1 participants were initially enrolled by age and in a dose-escalating design; each group included participants on active and placebo treatment in a 2:1 ratio. After Part 2 dose selection the study enrolled additional participants in a 2:1 ratio in Part 2.

Participant milestones

Participant milestones
Measure	Part 1 Group A Cohort 1: Adolescents and Adults (3 mg Risdiplam) Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks. Once the placebo-controlled period was completed and Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A Cohort 2: Adolescents and Adults (5 mg Risdiplam) Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks. Once the placebo-controlled period was completed and Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A Cohort 1: Adolescents and Adults (Placebo) Adolescent and adult participants aged 12-25 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to their cohort risdiplam dose (i.e. 3 mg). After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A Cohort 2: Adolescents and Adults (Placebo) Adolescent and adult participants aged 12-25 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to their cohort risdiplam dose (i.e. 5 mg). After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 1: Children (0.02 mg/kg Risdiplam) Children aged 2-11 years received risdiplam for at least 12 weeks. During the placebo-controlled period, participants escalated to 0.05 mg/kg and then to 0.15 mg/kg in two steps. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 2: Children (0.05 mg/kg Risdiplam) Children aged 2-11 years received risdiplam for at least 12 weeks. During the placebo-controlled period, participants escalated to 0.15 mg/kg in one step. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 3: Children (0.25 mg/kg Risdiplam) Children aged 2-11 years received risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 1: Children (Placebo) Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to the final 0.15 mg/kg cohort risdiplam dose. After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 2: Children (Placebo) Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to the final 0.15 mg/kg cohort risdiplam dose. After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 3: Children (Placebo) Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A: OLE Once the placebo-controlled period was completed and Part 2 dose was selected, adolescents and adults switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 1 Group B: OLE Once the placebo-controlled period was completed and Part 2 dose was selected, children switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 2: Risdiplam Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months. Once the Part 2 placebo-controlled period was completed participants received risdiplam at the same dose level for another 12 months (Month 12-24) in the open-label treatment (OLT) phase. After Month 24 participants entered the Part 2 OLE phase and continued to receive risdiplam at the same dose level.	Part 2: Placebo Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner in the OLT phase. After Month 24 participants entered the Part 2 OLE phase and continued to receive risdiplam at the same dose level.
Part 1 Placebo-Controlled STARTED	7	7	3	3	7	7	7	3	4	3	0	0	0	0
Part 1 Placebo-Controlled COMPLETED	7	7	3	3	7	7	7	3	4	3	0	0	0	0
Part 1 Placebo-Controlled NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Part 1 OLE STARTED	0	0	0	0	0	0	0	0	0	0	20	31	0	0
Part 1 OLE COMPLETED	0	0	0	0	0	0	0	0	0	0	18	30	0	0
Part 1 OLE NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	2	1	0	0
Part 2 Placebo-Controlled: Month 1-12 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	120	60
Part 2 Placebo-Controlled: Month 1-12 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	117	59
Part 2 Placebo-Controlled: Month 1-12 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	3	1
Part 2 OLT: Month 12-24 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	117	59
Part 2 OLT: Month 12-24 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	116	59
Part 2 OLT: Month 12-24 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	1	0
Part 2 OLE: > Month 24 STARTED	0	0	0	0	0	0	0	0	0	0	0	0	116	59
Part 2 OLE: > Month 24 COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	103	53
Part 2 OLE: > Month 24 NOT COMPLETED	0	0	0	0	0	0	0	0	0	0	0	0	13	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Part 1 Group A: OLE Once the placebo-controlled period was completed and Part 2 dose was selected, adolescents and adults switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 1 Group B: OLE Once the placebo-controlled period was completed and Part 2 dose was selected, children switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 2: Risdiplam Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months. Once the Part 2 placebo-controlled period was completed participants received risdiplam at the same dose level for another 12 months (Month 12-24) in the open-label treatment (OLT) phase. After Month 24 participants entered the Part 2 OLE phase and continued to receive risdiplam at the same dose level.	Part 2: Placebo Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner in the OLT phase. After Month 24 participants entered the Part 2 OLE phase and continued to receive risdiplam at the same dose level.
Part 1 OLE Withdrawal by Subject	2	1	0	0
Part 2 Placebo-Controlled: Month 1-12 Changed to Spinraza	0	0	2	1
Part 2 Placebo-Controlled: Month 1-12 Changed to other treatment	0	0	1	0
Part 2 OLT: Month 12-24 Withdrawal by Subject	0	0	1	0
Part 2 OLE: > Month 24 Death	0	0	1	0
Part 2 OLE: > Month 24 Reason not specified	0	0	2	1
Part 2 OLE: > Month 24 Withdrawal by Subject	0	0	10	5

Baseline Characteristics

Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part 1 Group A Cohort 1: Adolescents and Adults (3 mg Risdiplam) n=7 Participants Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks. Once the placebo-controlled period was completed and Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A Cohort 2: Adolescents and Adults (5 mg Risdiplam) n=7 Participants Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks. Once the placebo-controlled period was completed and Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A Cohort 1: Adolescents and Adults (Placebo) n=3 Participants Adolescent and adult participants aged 12-25 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to their cohort risdiplam dose (i.e. 3 mg). After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group A Cohort 2: Adolescents and Adults (Placebo) n=3 Participants Adolescent and adult participants aged 12-25 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to their cohort risdiplam dose (i.e. 5 mg). After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 1: Children (0.02 mg/kg Risdiplam) n=7 Participants Children aged 2-11 years received risdiplam for at least 12 weeks. During the placebo-controlled period, participants escalated to 0.05 mg/kg and then to 0.15 mg/kg in two steps. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 2: Children (0.05 mg/kg Risdiplam) n=7 Participants Children aged 2-11 years received risdiplam for at least 12 weeks. During the placebo-controlled period, participants escalated to 0.15 mg/kg in one step. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 3: Children (0.25 mg/kg Risdiplam) n=7 Participants Children aged 2-11 years received risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 1: Children (Placebo) n=3 Participants Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to the final 0.15 mg/kg cohort risdiplam dose. After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 2: Children (Placebo) n=4 Participants Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, participants first switched to the final 0.15 mg/kg cohort risdiplam dose. After the Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 1 Group B Cohort 3: Children (Placebo) n=3 Participants Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks. Once the placebo-controlled period was completed, and after Part 2 dose was selected, participants switched to Part 2 dose and were treated in an open-label phase.	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.	Total n=231 Participants Total of all reporting groups
Age, Continuous Part 1	13.3 Years STANDARD_DEVIATION 1.1 • n=7 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	18.1 Years STANDARD_DEVIATION 4.6 • n=7 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	14.7 Years STANDARD_DEVIATION 1.5 • n=3 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	17.3 Years STANDARD_DEVIATION 5.1 • n=3 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	6.1 Years STANDARD_DEVIATION 2.9 • n=7 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	4.3 Years STANDARD_DEVIATION 1.7 • n=7 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	6.0 Years STANDARD_DEVIATION 2.7 • n=7 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	5.3 Years STANDARD_DEVIATION 2.1 • n=3 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	3.5 Years STANDARD_DEVIATION 0.6 • n=4 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	5.3 Years STANDARD_DEVIATION 2.9 • n=3 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	—	—	9.4 Years STANDARD_DEVIATION 6.0 • n=51 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.
Age, Continuous Part 2	—	—	—	—	—	—	—	—	—	—	9.9 Years STANDARD_DEVIATION 5.8 • n=120 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	10.3 Years STANDARD_DEVIATION 6.0 • n=60 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.	10.0 Years STANDARD_DEVIATION 5.8 • n=180 Participants • Intent-to-Treat (ITT) population: Part 1 and Part 2 were analyzed separately and population results are provided for each part in the study.
Sex: Female, Male Female	5 Participants n=7 Participants	5 Participants n=7 Participants	1 Participants n=3 Participants	2 Participants n=3 Participants	5 Participants n=7 Participants	3 Participants n=7 Participants	4 Participants n=7 Participants	0 Participants n=3 Participants	2 Participants n=4 Participants	0 Participants n=3 Participants	61 Participants n=120 Participants	30 Participants n=60 Participants	118 Participants n=231 Participants
Sex: Female, Male Male	2 Participants n=7 Participants	2 Participants n=7 Participants	2 Participants n=3 Participants	1 Participants n=3 Participants	2 Participants n=7 Participants	4 Participants n=7 Participants	3 Participants n=7 Participants	3 Participants n=3 Participants	2 Participants n=4 Participants	3 Participants n=3 Participants	59 Participants n=120 Participants	30 Participants n=60 Participants	113 Participants n=231 Participants
Race/Ethnicity, Customized Asian	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=3 Participants	1 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	23 Participants n=120 Participants	12 Participants n=60 Participants	36 Participants n=231 Participants
Race/Ethnicity, Customized White	7 Participants n=7 Participants	7 Participants n=7 Participants	3 Participants n=3 Participants	3 Participants n=3 Participants	6 Participants n=7 Participants	6 Participants n=7 Participants	7 Participants n=7 Participants	3 Participants n=3 Participants	3 Participants n=4 Participants	3 Participants n=3 Participants	80 Participants n=120 Participants	41 Participants n=60 Participants	169 Participants n=231 Participants
Race/Ethnicity, Customized Multiple	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=7 Participants	1 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	1 Participants n=4 Participants	0 Participants n=3 Participants	1 Participants n=120 Participants	0 Participants n=60 Participants	3 Participants n=231 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	2 Participants n=120 Participants	0 Participants n=60 Participants	2 Participants n=231 Participants
Race/Ethnicity, Customized Unknown	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	1 Participants n=120 Participants	0 Participants n=60 Participants	1 Participants n=231 Participants
Race/Ethnicity, Customized Hispanic or Latino	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=3 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	5 Participants n=120 Participants	2 Participants n=60 Participants	7 Participants n=231 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	7 Participants n=7 Participants	7 Participants n=7 Participants	3 Participants n=3 Participants	3 Participants n=3 Participants	6 Participants n=7 Participants	7 Participants n=7 Participants	7 Participants n=7 Participants	3 Participants n=3 Participants	4 Participants n=4 Participants	3 Participants n=3 Participants	114 Participants n=120 Participants	57 Participants n=60 Participants	221 Participants n=231 Participants
Race/Ethnicity, Customized Not Stated	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=3 Participants	1 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=7 Participants	0 Participants n=3 Participants	0 Participants n=4 Participants	0 Participants n=3 Participants	0 Participants n=120 Participants	1 Participants n=60 Participants	2 Participants n=231 Participants

PRIMARY outcome

Timeframe: Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017)

Population: All participants in Part 1.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=51 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 1: Selected Part 2 Dose of Risdiplam for Participants With a Body Weight (BW) of >/=20kg	5 milligram (mg)	—

PRIMARY outcome

Timeframe: Day 1 up to at least 4 weeks on study (Up to CCOD of 25 July 2017)

Population: All participants in Part 1.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=51 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 1: Selected Part 2 Dose of Risdiplam for Participants With BW of <20kg	0.25 milligram/kilogram (mg/kg)	—

PRIMARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis.

The Motor Function Measure 32 (MFM32) is a scale constructed for use in neuromuscular disorders. It comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The 32 scores are summed and expressed on a 0-100 scale for the MFM32 total score. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on primary efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=115 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Total Motor Function Measure 32 (MFM-32) Total Score at Month 12	1.36 Scores on a Scale Interval 0.61 to 2.11	-0.19 Scores on a Scale Interval -1.22 to 0.84

SECONDARY outcome

Timeframe: At Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis. Missing results at Month 12 are considered as non-responders.

The MFM32 comprises 32 items that evaluate physical function. The scoring of each task uses a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The 32 scores are summed and expressed on a 0-100 scale for the MFM32 total score. A change in MFM32 total score of threshold \>/=3 represents marked improvement in this measure. Logistic regression analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=115 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants With Marked Improvement (Defined as >= 3) in the Total Motor Function Measure (MFM32) Score at Month 12	38.3 Percentage of Participants Interval 28.94 to 47.58	23.7 Percentage of Participants Interval 12.03 to 35.43

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing RULM total score at Baseline were not included in the analysis.

The RULM is a 20 items scale that assesses the proximal and distal motor functions of the arm. There is an entry item and the remaining 18 items are scored on the 3 point scale of: 0: cannot complete task independently; 1: modified method but can complete task independently; 2: completes task without any assistance, and with 1 item scored on a 2 point scale of as a can/cannot score with 1 as the highest score. The RULM total score is the sum of 19 items scores with range of 0-37, and the entry item does not contribute to the total score. Higher scores indicate greater upper limb function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on the efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=119 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=58 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Total Score of the Revised Upper Limb Module (RULM) at Month 12	1.61 Scores on a Scale Interval 1.0 to 2.22	0.02 Scores on a Scale Interval -0.83 to 0.87

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2.

The HFMSE scale contains 33 items, which are scored on a 3-point Likert-type scale (0-2) and summed to derive the total score, with lower scores indicating greater impairment. The HFMSE contains a series of assessments designed to assess important functional abilities, including standing, transfers, ambulation, and proximal and axial function. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate greater motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on the efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in Total Score of Hammersmith Functional Motor Scale Expanded (HFMSE) at Month 12	0.95 Scores on a Scale Interval 0.29 to 1.61	0.37 Scores on a Scale Interval -0.54 to 1.28

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing FVC data at Baseline were not included in the analysis.

Spirometry is a pulmonary function test that assesses how the lungs work by measuring how much air moves through the airways. Spirometry was performed by all participants aged 6 or older. Forced vital capacity (FVC) is the total volume that can be exhaled after inhaling maximally. The best % predicted value out of all attempts were used for the analysis. MMRM analysis was performed based on the efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=83 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=40 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in Forced Vital Capacity (FVC) at Month 12 in Participants Aged 6-25 Years	-5.16 Percentage Predicted Interval -7.93 to -2.39	-3.11 Percentage Predicted Interval -6.59 to 0.74

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing SMAIS total score at Baseline were not included in the analysis.

The SMA Independence Scale (SMAIS) was developed specifically for SMA participants in order to assess function-related independence. The SMAIS contains 29 items, assessing the amount of assistance required from another individual to perform daily activities such as eating, or bathing. Each item is scored on a 0-4 scale (with an additional option to indicate that an item is non-applicable). The SMAIS total score ranging from 0-44 is obtained based on 22 items with each item on the 0-2 scale. Lower scores indicate greater dependence on another individual. The SMAIS was completed by participants aged 12 years or older and caregivers of participants aged 2-25 years. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=116 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Caregiver-Reported SMA Independence Scale (SMAIS) Total Score at Month 12	1.65 Scores on a Scale Interval 0.66 to 2.63	-0.91 Scores on a Scale Interval -2.23 to 0.42

SECONDARY outcome

Timeframe: At Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Missing results at Month 12 are considered as non-responders.

The Clinical Global Impression of Change (CGI-C) is used to score a clinician's impression of a participant's change in global health. The CGI-C is a single item measure of change in global health, using seven response options: "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", and "very much worse". Participants considered as "improved" included responses of "very much improved, "much improved" and "minimally improved". Logistic regression analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants Rated by Clinicians as Improved in the Clinical Global Impression of Change (CGI-C) Scale Ratings at Month 12	47.5 Percentage of Participants Interval 38.15 to 56.86	40.0 Percentage of Participants Interval 26.77 to 53.23

SECONDARY outcome

Timeframe: At Month 12

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The 32 scores are summed and expressed on a 0-100 scale for the MFM32 total score. Higher scores indicate increased motor function. Logistic regression analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=115 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants Who Achieve Stabilization or Improvement (Defined as >= 0) in the Total Motor Function Measure (MFM-32) Score at Month 12	69.6 Percentage of Participants Interval 60.72 to 78.41	54.2 Percentage of Participants Interval 40.68 to 67.8

SECONDARY outcome

Timeframe: At Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 total score at Baseline were not included in the analysis.

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0-cannot initiate the task or maintain the starting position; 1-performs the task partially; 2-performs the task incompletely or imperfectly; 3-performs the task fully and "normally". The 32 scores are summed and expressed on a 0-100 scale for the total score. Higher scores indicate increased motor function. Standard error of measurement (SEM) is derived using 32 items scores and total scores at baseline. Change from baseline \> = one SEM is equivalent to a change \>= 4. Logistic regression analysis was performed based on efficacy hypothetical estimand included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=115 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants Who Achieve an Improvement of at Least One Standard Error of Measurement on the Total MFM-32 Score at Month 12	28.7 Percentage of Participants Interval 20.65 to 37.88	16.9 Percentage of Participants Interval 8.44 to 28.97

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D1 score at Baseline were not included in the analysis.

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The D1 items score are summed and expressed on 0-100 scale for the MFM D1 total score. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=118 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the MFM-32 Domain 1 (D1) Score at Month 12	0.37 Scores on a Scale Interval -0.12 to 0.87	-0.26 Scores on a Scale Interval -0.94 to 0.42

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D2 score at Baseline were not included in the analysis.

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The D2 items score are summed and expressed on 0-100 scale for the MFM D2 total score. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=118 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the MFM-32 Domain 2 (D2) Score at Month 12	1.04 Scores on a Scale Interval -0.38 to 2.46	-0.93 Scores on a Scale Interval -2.87 to 1.02

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D3 score at Baseline were not included in the analysis.

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The D3 items score are summed and expressed on 0-100 scale for the MFM D3 total score. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=115 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the MFM-32 Domain 3 (D3) Score at Month 12	3.68 Scores on a Scale Interval 2.31 to 5.04	1.34 Scores on a Scale Interval -0.54 to 3.22

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D1+D2 scores at Baseline were not included in the analysis.

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The D1+D2 items score are summed and expressed on 0-100 scale for the MFM D1+D2 total score. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=118 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Total Combined Scores of MFM-32 Domains 1 and 2 at Month 12	0.69 Scores on a Scale Interval -0.07 to 1.45	-0.59 Scores on a Scale Interval -1.64 to 0.45

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MFM32 D2+D3 scores at Baseline were not included in the analysis.

The MFM32 comprises 32 items that evaluate physical function in three dimensions: D1 function related to standing and transfer; D2 axial and proximal function; D3 distal motor function. Tasks are scored with a 4-point Likert scale: 0 - cannot initiate the task or maintain the starting position; 1 - performs the task partially; 2 - performs the task incompletely or imperfectly; 3 - performs the task fully and "normally". The D2+D3 items score are summed and expressed on 0-100 scale for the MFM D2+D3 total score. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=115 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Total Combined Scores of MFM-32 Domains 2 and 3 at Month 12	2.02 Scores on a Scale Interval 0.84 to 3.2	-0.14 Scores on a Scale Interval -1.76 to 1.48

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing FEV1 data at Baseline were not included in the analysis.

Spirometry is a pulmonary function test that assesses how the lungs work by measuring how much air moves through the airways. Spirometry was performed by all participants aged 6 or older. Forced expiratory volume (FEV1) is the volume forcefully exhaled in the first second of the forced vital capacity test. The best % predicted value out of all attempts were used for the analysis. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=83 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=40 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Month 12 in Participants Aged 6-25 Years	-4.22 Percentage Predicted Interval -7.49 to -0.96	-1.35 Percentage Predicted Interval -5.91 to 3.2

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing PCF data at Baseline were not included in the analysis.

Spirometry is a pulmonary function test that assesses how the lungs work by measuring how much air moves through the airways. Spirometry was performed by all participants aged 6 or older. Peak cough flow (PCF) is an assessment of cough strength. The best % predicted value out of all attempts were used for the analysis MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=83 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=42 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Peak Cough Flow (PCF) at Month 12 in Participants Aged 6-25 Years	1.06 Percent Predicted Interval -1.18 to 3.31	-0.22 Percent Predicted Interval -3.27 to 2.83

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing SNIP data at Baseline were not included in the analysis.

The Sniff Nasal Inspiratory Pressure (SNIP) is a volitional, non-invasive test of inspiratory muscle strength that has been successfully applied to children \> 2 years of age. Advantages include the simplicity of the maneuver and the absence of a mouthpiece, which is particularly helpful for participants with SMA, who may have bulbar weakness. SNIP also has the advantage of measuring inspiratory pressure during a natural maneuver that is easily performed even by young children with neuromuscular disorders. The best % predicted value out of all attempts were used for the analysis. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=118 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=59 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Best Sniff Nasal Inspiratory Pressure (SNIP) at Month 12	3.42 Percentage Predicted Interval 0.22 to 6.62	1.07 Percentage Predicted Interval -3.42 to 5.57

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MIP data at Baseline were not included in the analysis.

The maximal inspiratory pressure (MIP) is a non-invasive test of muscle strength, which measures the maximum strength of the diaphragm and other inspiratory muscles. MIP was measured in participants aged 6 or older. Participants were asked to perform a forceful inspiration against an occluded mouth piece. The best % predicted value out of all attempts were used for the analysis. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=81 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=40 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in Maximal Inspiratory Pressure (MIP) at Month 12 in Participants Aged 6-25 Years	1.99 Percentage Predicted Interval -6.13 to 10.11	-0.97 Percentage Predicted Interval -12.33 to 10.38

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing MEP data at Baseline were not included in the analysis.

The maximal expiratory pressure (MEP) is a non-invasive test of muscle strength, which measures the maximum strength of the abdominal muscles and other expiratory muscles. MEP was measured in participants aged 6 or older. Participants were asked to perform a forceful inspiration against an occluded mouth piece. The best % predicted value out of all attempts were used for the analysis. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=83 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=41 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in Maximal Expiratory Pressure (MEP) at Month 12 in Participants Aged 6-25 Years	-2.75 Percentage Predicted Interval -6.22 to 0.72	-2.33 Percentage Predicted Interval -7.21 to 2.56

SECONDARY outcome

Timeframe: Baseline (Day-1) and Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Participants with missing SMAIS total score at Baseline were not included in the analysis.

The SMAIS was developed specifically for SMA participants in order to assess function-related independence. It contains 29 items, assessing the amount of assistance required from another individual to perform daily activities such as eating, or bathing. Each item is scored on a 0-4 scale (with an additional option to indicate that an item is non-applicable). The SMAIS total score ranging from 0-44 is obtained based on 22 items with each item on the 0-2 scale. Lower scores indicate greater dependence on another individual. The SMAIS was completed by participants aged 12 years or older and caregivers of participants aged 2-25 years. MMRM analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=43 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=23 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Change From Baseline in the Participant-Reported SMA Independence Scale (SMAIS) Total Score at Month 12	1.04 Scores on a Scale Interval -0.26 to 2.35	-0.40 Scores on a Scale Interval -2.13 to 1.32

SECONDARY outcome

Timeframe: At Month 12

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Missing results at Month 12 are considered as non-responders.

The CGI-C is used to score a clinician's impression of a participant's change in global health. It is a single item measure of change in global health, using seven response options: "very much improved", "much improved", "minimally improved", "no change", "minimally worse", "much worse", and "very much worse". Participants considered as "no change or improved" included responses of "no change", "very much improved", "much improved" and "minimally improved". Logistic regression analysis was performed based on efficacy hypothetical estimand, which included participants data assuming no prohibited medication intended for treatment of SMA was received and participants continued on their randomized treatment until the analysis time point at Month 12.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants Rated by Clinicians as No Change or Improved in the Clinical Global Impression of Change (CGI-C) Scale Ratings at Month 12	85.8 Percentage of Participants Interval 79.18 to 92.49	83.3 Percentage of Participants Interval 73.07 to 93.6

SECONDARY outcome

Timeframe: Baseline up to Month 12 (Week 52; up to CCOD of 06 September 2019)

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2.

Disease-related adverse events (AEs) were identified by applying two different types of baskets to the AE dataset: Narrow prospectively defined baskets of MedDRA lowest level terms. This basket was defined based on a group of CDC terms selected from an age and gender matched case control study comparing CDC code rates observed in participants with and without SMA using commercially available insurance claim data (CLAIMS and Market scan data). The lowest level terms included in each basket, coded using the latest version of MedDRA; Broad prospectively defined basket with events selected at preferred term level from all AEs reported in ongoing clinical trials up to January 2019, i.e., prior to unblinding of Part 2 of Study BP39055.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants Who Experience at Least One Disease-Related Adverse Event at Month 12 Broad Basket AEs	65.0 Percentage of Participants Interval 55.76 to 73.48	60.0 Percentage of Participants Interval 46.54 to 72.44
Part 2: Percentage of Participants Who Experience at Least One Disease-Related Adverse Event at Month 12 Narrow Basket AEs	46.7 Percentage of Participants Interval 37.51 to 55.99	53.3 Percentage of Participants Interval 40.0 to 66.33

SECONDARY outcome

Timeframe: Baseline up to Month 12 (Week 52; up to CCOD of 06 September 2019)

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2.

Disease-related AEs were collected through the AE reporting of the study, and the disease-related AE rate was adjusted for patient years (AE rate per 100 patient-years). They were identified by applying two different types of baskets to the AE dataset: Narrow prospectively defined baskets of MedDRA lowest level terms and Broad prospectively defined basket with events selected at preferred term level from all AEs reported in ongoing clinical trials up to January 2019, i.e., prior to unblinding of Part 2 of Study BP39055.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Number of Disease-related Adverse Events Per Patient-years at Month 12 Narrow Basket AEs	101.51 Number of Events per 100 Patient-Years Interval 84.23 to 121.29	119.77 Number of Events per 100 Patient-Years Interval 93.71 to 150.82
Part 2: Number of Disease-related Adverse Events Per Patient-years at Month 12 Broad Basket AEs	217.29 Number of Events per 100 Patient-Years Interval 191.63 to 245.42	199.61 Number of Events per 100 Patient-Years Interval 165.5 to 238.68

SECONDARY outcome

Timeframe: Day 1 up to 12 months of the placebo-controlled period

Population: All participants in Part 2 who receive at least one dose of study medication (risdiplam or placebo) were included in the safety population.

An adverse event (AE) is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in the Placebo-Controlled Period With at Least One AE	92.5 Percentage of Participants	91.7 Percentage of Participants
Part 2: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in the Placebo-Controlled Period With at Least One SAE	20.0 Percentage of Participants	18.3 Percentage of Participants

SECONDARY outcome

Timeframe: Day 1 up to 12 months of the placebo-controlled period

Population: All participants in Part 2 who received at least one dose of study medication (risdiplam or placebo) were included in the safety population.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=120 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=60 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Percentage of Participants With Treatment Discontinuation Due to Adverse Events (AEs) and Serious Adverse Events (SAEs) in the Placebo-Controlled Period Due to AE	0.0 Percentage of Participants	0.0 Percentage of Participants
Part 2: Percentage of Participants With Treatment Discontinuation Due to Adverse Events (AEs) and Serious Adverse Events (SAEs) in the Placebo-Controlled Period Due to SAE	0.0 Percentage of Participants	0.0 Percentage of Participants

SECONDARY outcome

Timeframe: Day 1 up to 12 months of the placebo-controlled period

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Data was collected for participants aged 6-25 years.

The Columbia Suicide Severity Rating Scale (C-SSRS) is used to assess the lifetime suicidality of a participant (C-SSRS baseline) as well as any new instances of suicidality (C-SSRS since last visit). The structured interview prompts recollection of suicidal ideation, including the intensity of the ideation, behavior, and attempts with actual/potential lethality. The C-SSRS assessments results were collected for participants aged 6 years and older.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=83 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=42 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Number of Participants Aged 6-25 Years With Suicidal Ideation Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Wish to be Dead	1 Number of Participants	1 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Ideation Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Non-specific Active Suicidal Thoughts	1 Number of Participants	1 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Ideation Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Ideation with Any Methods, No Intent to Act	1 Number of Participants	1 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Ideation Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Ideation with Some Intent to Act, No Plan	0 Number of Participants	1 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Ideation Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Ideation with Specific Plan and Intent	0 Number of Participants	1 Number of Participants

SECONDARY outcome

Timeframe: Day 1 up to 12 months of the placebo-controlled period

Population: The intent-to-treat (ITT) population defined as all randomized participants in Part 2. Data was collected for participants aged 6-25 years.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=83 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=42 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 2: Number of Participants Aged 6-25 Years With Suicidal Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Preparatory Acts or Behavior	0 Number of Participants	0 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Aborted Attempt	0 Number of Participants	0 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Interrupted Attempt	0 Number of Participants	0 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Actual Attempt (non-fatal)	0 Number of Participants	0 Number of Participants
Part 2: Number of Participants Aged 6-25 Years With Suicidal Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) in the Placebo-Controlled Period Completed Suicide	0 Number of Participants	0 Number of Participants

SECONDARY outcome

Timeframe: Part 1: Day -1, pre-dose of Weeks 1, 2 (>/= 12 years only), 17, 35 and 104, and at 4h post-dose of Weeks 4 and 52. Part 2: Day -1, pre-dose of Weeks 1, 17, 35 and 104, and at 4h post-dose of Weeks 4 and 52.

Population: All participants with at least one time point with a protein measurement were included in the pharmacodynamic (PD) analysis data set.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=51 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=169 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Median Fold Change From Baseline in Survival of Motor Neuron (SMN) Protein Levels in Blood	2.91 unitless Interval 2.14 to 4.18	1.96 unitless Interval 0.2 to 4.48

SECONDARY outcome

Timeframe: Day 1: 1, 2, 4, 6 h postdose, Weeks 4, 8 (Part 1 only), 52, 87: pre-dose, 1, 2, 4, 6 h post-dose and Weeks 1 (Day 7), 2, 8 (Part 2 only) 17, 35, 70, 104: predose

Population: All participants with at least one time point with a risdiplam concentration measurement were included in the pharmacokinetic (PK) analysis data set.

Reported here is the maximum observed concentration throughout the observation period.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=51 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=179 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 1 and 2: Maximum Plasma Concentration (Cmax) of Risdiplam at Year 5	137 nanograms/milliliter (ng/mL) Interval 58.2 to 242.0	140 nanograms/milliliter (ng/mL) Interval 42.7 to 313.0

SECONDARY outcome

Timeframe: Year 5 visit pre-dose, 1, 2, 4, 6, 24 hours post-dose

Population: All participants with at least one time point with a risdiplam concentration measurement were included in the PK analysis data set.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=49 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=160 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 1 and 2: Area Under the Curve (AUC) From 0 to 24 Hours of Risdiplam at Year 5 Visit	1700 ng*h/mL Interval 1160.0 to 2590.0	1880 ng*h/mL Interval 1200.0 to 2890.0

SECONDARY outcome

Timeframe: The last predose sample collected from each participant who had at least 1400 days of risdiplam treatment duration.

Population: All participants with at least one time point with a risdiplam concentration measurement were included in the PK analysis data set.

Outcome measures

Outcome measures
Measure	Part 2: Risdiplam n=49 Participants Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months.	Part 2: Placebo n=107 Participants Participants aged 2-25 years received placebo matching to risdiplam for 12 months. After 12 months of treatment with placebo, participants switched to risdiplam (5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20) in a blinded manner and participants will continue with treatment and observations.
Part 1 and 2: Concentration at the End of a Dosing Interval (Ctrough) of Risdiplam at Year 5	54.1 ng/mL Interval 21.3 to 108.0	57.2 ng/mL Interval 4.5 to 229.0

Adverse Events

Part 1 Group A: Adolescents and Adults (3 mg Risdiplam)

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

Part 1 Group A: Adolescents and Adults (5 mg Risdiplam)

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Part 1 Group A: Adolescents and Adults (Placebo-Control Period Pooled)

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Part 1 Group B: Children (0.02 mg/kg Risdiplam)

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Part 1 Group B: Children (0.05 mg/kg Risdiplam)

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Part 1 Group B: Children (0.15 mg/kg Risdiplam)

Serious events: 0 serious events

Other events: 18 other events

Deaths: 0 deaths

Part 1 Group B: Children (0.25 mg/kg Risdiplam)

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Part 1 Group B: Children (Placebo-Control Period Pooled)

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

Part 1 Group A: OLE

Serious events: 5 serious events

Other events: 17 other events

Deaths: 0 deaths

Part 1 Group B: OLE

Serious events: 9 serious events

Other events: 28 other events

Deaths: 0 deaths

Part 2 Placebo-Controlled: Risdiplam

Serious events: 24 serious events

Other events: 101 other events

Deaths: 0 deaths

Part 2 Placebo-Controlled: Placebo

Serious events: 11 serious events

Other events: 50 other events

Deaths: 0 deaths

Part 2 OLT: Risdiplam/Risdiplam

Serious events: 25 serious events

Other events: 88 other events

Deaths: 0 deaths

Part 2 OLT: Placebo/Risdiplam

Serious events: 4 serious events

Other events: 40 other events

Deaths: 0 deaths

Part 2 OLE: Risdiplam

Serious events: 34 serious events

Other events: 140 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Part 1 Group A: Adolescents and Adults (3 mg Risdiplam) n=10 participants at risk Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group A: Adolescents and Adults (5 mg Risdiplam) n=10 participants at risk Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group A: Adolescents and Adults (Placebo-Control Period Pooled) n=6 participants at risk Adolescent and adult participants aged 12-25 years received placebo matching to risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.02 mg/kg Risdiplam) n=7 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.05 mg/kg Risdiplam) n=14 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.15 mg/kg Risdiplam) n=21 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.25 mg/kg Risdiplam) n=7 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (Placebo-Control Period Pooled) n=10 participants at risk Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group A: OLE n=20 participants at risk Once the placebo-controlled period was completed and Part 2 dose was selected, adolescents and adults switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 1 Group B: OLE n=31 participants at risk Once the placebo-controlled period was completed and Part 2 dose was selected, children switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 2 Placebo-Controlled: Risdiplam n=120 participants at risk Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months during the placebo-controlled period..	Part 2 Placebo-Controlled: Placebo n=60 participants at risk Participants aged 2-25 years received placebo matching to risdiplam for 12 months during the placebo-controlled period.	Part 2 OLT: Risdiplam/Risdiplam n=117 participants at risk Once the Part 2 placebo-controlled period was completed participants received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for another 12 months (Month 12-24) in the open-label treatment (OLT) phase.	Part 2 OLT: Placebo/Risdiplam n=59 participants at risk Once the Part 2 placebo-controlled period was completed participants switched to risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months (Month 12-24) in the open-label treatment (OLT) phase.	Part 2 OLE: Risdiplam n=175 participants at risk Once the Part 2 OLT period ended participants entered the open-label extension period and continued to receive risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg.
Infections and infestations Respiratory tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Abdominal pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Oesophagitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Vomiting	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Appendicitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Gastroenteritis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Influenza	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pneumonia	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.3% 10/120 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.8% 8/117 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.6% 8/175 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pneumonia mycoplasmal	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Upper respiratory tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Concussion	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Femur fracture	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Near drowning	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Decreased appetite	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Dehydration	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Chronic respiratory failure	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Blood and lymphatic system disorders Spontaneous haematoma	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Congenital, familial and genetic disorders Cryptorchism	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Colitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Constipation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Dental caries	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Duodenal obstruction	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Gastritis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Gastrointestinal haemorrhage	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Gastrooesophageal reflux disease	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Malpositioned teeth	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Ill-defined disorder	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Medical device pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Pyrexia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Hepatobiliary disorders Hypertransaminasaemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Adenovirus infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Bacteraemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Bronchitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations COVID-19	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations COVID-19 pneumonia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Device related infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Encephalitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Gastrointestinal infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Helicobacter infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Herpes zoster	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Infective thrombosis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Laryngitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Lower respiratory tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Lower respiratory tract infection viral	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Lymph gland infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pneumonia aspiration	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pneumonia bacterial	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Post procedural infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Postoperative wound infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pyelonephritis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Respiratory tract infection viral	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Skin infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Tonsillitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Viral upper respiratory tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Wound infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Brain contusion	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Fall	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Musculoskeletal procedural complication	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Toxicity to various agents	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Wound dehiscence	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Investigations Oxygen saturation decreased	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Hypoglycaemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Hypokalaemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Metabolic acidosis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Nervous system disorders Febrile convulsion	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Nervous system disorders Partial seizures	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Nervous system disorders Syncope	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Product Issues Device breakage	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Psychiatric disorders Affective disorder	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Psychiatric disorders Encopresis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Renal and urinary disorders Haematuria	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Renal and urinary disorders Hydronephrosis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Renal and urinary disorders Nephrolithiasis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Renal and urinary disorders Renal colic	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Aspiration	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Asthma	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Atelectasis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Obstructive sleep apnoea syndrome	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Pneumonitis aspiration	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Pneumothorax	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Respiratory disorder	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Vascular disorders Haematoma	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Vascular disorders Superficial vein thrombosis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.

Other adverse events

Other adverse events
Measure	Part 1 Group A: Adolescents and Adults (3 mg Risdiplam) n=10 participants at risk Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group A: Adolescents and Adults (5 mg Risdiplam) n=10 participants at risk Adolescent and adult participants aged 12-25 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group A: Adolescents and Adults (Placebo-Control Period Pooled) n=6 participants at risk Adolescent and adult participants aged 12-25 years received placebo matching to risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.02 mg/kg Risdiplam) n=7 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.05 mg/kg Risdiplam) n=14 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.15 mg/kg Risdiplam) n=21 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (0.25 mg/kg Risdiplam) n=7 participants at risk Children aged 2-11 years received risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group B: Children (Placebo-Control Period Pooled) n=10 participants at risk Children aged 2-11 years received placebo matching to risdiplam for at least 12 weeks during the placebo-controlled period.	Part 1 Group A: OLE n=20 participants at risk Once the placebo-controlled period was completed and Part 2 dose was selected, adolescents and adults switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 1 Group B: OLE n=31 participants at risk Once the placebo-controlled period was completed and Part 2 dose was selected, children switched to Part 2 dose and were treated in an open-label extension (OLE) phase.	Part 2 Placebo-Controlled: Risdiplam n=120 participants at risk Participants aged 2-25 years received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months during the placebo-controlled period..	Part 2 Placebo-Controlled: Placebo n=60 participants at risk Participants aged 2-25 years received placebo matching to risdiplam for 12 months during the placebo-controlled period.	Part 2 OLT: Risdiplam/Risdiplam n=117 participants at risk Once the Part 2 placebo-controlled period was completed participants received risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for another 12 months (Month 12-24) in the open-label treatment (OLT) phase.	Part 2 OLT: Placebo/Risdiplam n=59 participants at risk Once the Part 2 placebo-controlled period was completed participants switched to risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg for 12 months (Month 12-24) in the open-label treatment (OLT) phase.	Part 2 OLE: Risdiplam n=175 participants at risk Once the Part 2 OLT period ended participants entered the open-label extension period and continued to receive risdiplam at the dose of 5 mg once daily for participants with a body weight (BW) \>/=20kg or 0.25 mg/kg for participants with a BW \<20 kg.
Infections and infestations Scarlet fever	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Skin infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Blood and lymphatic system disorders Iron deficiency anaemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Blood and lymphatic system disorders Neutropenia	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Cardiac disorders Palpitations	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Cardiac disorders Sinus tachycardia	— 0/0 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Cardiac disorders Supraventricular tachycardia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Cardiac disorders Tachycardia	30.0% 3/10 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Ear and labyrinth disorders Ear pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.9% 4/31 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.3% 5/117 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Ear and labyrinth disorders Motion sickness	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Ear and labyrinth disorders Vertigo	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Eye disorders Conjunctival hyperaemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Eye disorders Corneal infiltrates	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Eye disorders Eczema eyelids	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Eye disorders Ocular hyperaemia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Eye disorders Retinal dystrophy	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Eye disorders Vision blurred	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Abdominal pain	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.9% 4/31 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.7% 8/120 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.3% 5/60 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.0% 7/117 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.8% 4/59 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.0% 7/175 • Number of events 15 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.8% 7/120 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 9/175 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Aphthous ulcer	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Constipation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.9% 4/31 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.5% 9/120 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 4/117 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.6% 8/175 • Number of events 9 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Diarrhoea	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	17.5% 21/120 • Number of events 27 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.3% 5/60 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.5% 10/117 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.2% 6/59 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 17/175 • Number of events 26 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Faecaloma	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Gastrointestinal disorder	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Lip pruritus	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Nausea	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.5% 2/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.2% 11/120 • Number of events 13 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 4/117 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 3/59 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.6% 8/175 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Oral mucosal erythema	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Swollen tongue	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Tongue oedema	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Toothache	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Gastrointestinal disorders Vomiting	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.6% 2/7 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.6% 4/14 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	23.8% 5/21 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 4/20 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	32.3% 10/31 • Number of events 27 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.2% 17/120 • Number of events 35 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	23.3% 14/60 • Number of events 22 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.8% 15/117 • Number of events 31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.3% 9/59 • Number of events 13 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	11.4% 20/175 • Number of events 43 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Asthenia	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Catheter site extravasation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Fatigue	20.0% 2/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.3% 4/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Granuloma	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Hyperpyrexia	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Hyperthermia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Influenza like illness	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 6/175 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Oedema	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
General disorders Pyrexia	20.0% 2/10 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.6% 2/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	21.4% 3/14 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	19.0% 4/21 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	30.0% 3/10 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	45.0% 9/20 • Number of events 19 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	58.1% 18/31 • Number of events 52 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.8% 25/120 • Number of events 41 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 10/60 • Number of events 20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.8% 15/117 • Number of events 22 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.2% 6/59 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	17.1% 30/175 • Number of events 38 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Immune system disorders Hypersensitivity	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.3% 4/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Bronchitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	22.6% 7/31 • Number of events 15 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.8% 7/120 • Number of events 9 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 10/60 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 6/117 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.8% 4/59 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 9/175 • Number of events 11 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations COVID-19	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	29.0% 9/31 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	31.4% 55/175 • Number of events 60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Conjunctivitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 4/120 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Cystitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Ear infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Ear infection fungal	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Gastroenteritis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 4/20 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	29.0% 9/31 • Number of events 11 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.8% 7/120 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.3% 5/60 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.7% 9/117 • Number of events 9 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.5% 5/59 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.9% 12/175 • Number of events 18 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Gastroenteritis viral	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 4/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Gastrointestinal infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Groin infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Influenza	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 4/20 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	19.4% 6/31 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.6% 8/175 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Laryngitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Laryngitis viral	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Nasopharyngitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.5% 2/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	38.7% 12/31 • Number of events 23 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	25.8% 31/120 • Number of events 54 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	25.0% 15/60 • Number of events 21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	22.2% 26/117 • Number of events 34 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.2% 6/59 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	19.4% 34/175 • Number of events 48 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pharyngitis	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.6% 2/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 2/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.5% 2/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.9% 4/31 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 6/120 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 6/117 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.8% 4/59 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.0% 7/175 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Pneumonia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.2% 5/120 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.9% 12/175 • Number of events 16 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Respiratory tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 2/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 3/21 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.6% 2/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.7% 8/120 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 6/60 • Number of events 9 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.0% 7/117 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.9% 5/175 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Rhinitis	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.2% 5/120 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 3/59 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.0% 7/175 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Tonsillitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.9% 5/175 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Tooth abscess	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Upper respiratory tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 2/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	19.0% 4/21 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.6% 2/7 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	45.2% 14/31 • Number of events 47 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	31.7% 38/120 • Number of events 54 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	30.0% 18/60 • Number of events 27 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.4% 18/117 • Number of events 24 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.9% 10/59 • Number of events 18 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	28.0% 49/175 • Number of events 99 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Urinary tract infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 6/120 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.9% 12/175 • Number of events 21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Viral infection	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.3% 4/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Sinusitis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 4/120 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.5% 5/59 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.9% 5/175 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Infections and infestations Varicella	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Arthropod bite	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 2/14 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.5% 2/21 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Contusion	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.2% 5/120 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Face injury	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Fall	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.3% 4/175 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Femur fracture	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Foot fracture	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Ligament sprain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Limb injury	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Injury, poisoning and procedural complications Tooth dislocation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Investigations Gastric pH decreased	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Investigations Hepatic enzyme increased	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Decreased appetite	10.0% 1/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Iron deficiency	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Metabolism and nutrition disorders Vitamin D deficiency	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.1% 5/31 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 6/120 • Number of events 9 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 2/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.4% 13/175 • Number of events 21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 4/20 • Number of events 14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.3% 11/175 • Number of events 13 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Myalgia	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Neck pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 13 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Osteoporosis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.9% 4/31 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 4/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 3/59 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.7% 10/175 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Nervous system disorders Dizziness	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.0% 7/175 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Nervous system disorders Headache	10.0% 1/10 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 18 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 18 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.5% 2/21 • Number of events 20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	19.4% 6/31 • Number of events 47 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 24/120 • Number of events 88 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 10/60 • Number of events 23 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.3% 12/117 • Number of events 76 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	18.6% 11/59 • Number of events 32 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	11.4% 20/175 • Number of events 84 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Psychiatric disorders Depression	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Psychiatric disorders Initial insomnia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Renal and urinary disorders Urinary tract pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Reproductive system and breast disorders Amenorrhoea	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Reproductive system and breast disorders Dysmenorrhoea	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 2/10 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 4/20 • Number of events 27 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Reproductive system and breast disorders Heavy menstrual bleeding	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Reproductive system and breast disorders Premenstrual pain	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Cough	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 2/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 3/21 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 2/10 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 4/20 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	35.5% 11/31 • Number of events 31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.2% 17/120 • Number of events 27 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 12/60 • Number of events 19 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.3% 12/117 • Number of events 16 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.5% 5/59 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	8.0% 14/175 • Number of events 19 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Lower respiratory tract inflammation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Nasal congestion	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.5% 2/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	20.0% 2/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	30.0% 3/10 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	19.4% 6/31 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 6/120 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	11.7% 7/60 • Number of events 8 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.4% 4/117 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	6.3% 11/175 • Number of events 16 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Pleurisy	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Respiratory tract inflammation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 2/20 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.3% 2/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 3/175 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 6/120 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.7% 10/175 • Number of events 12 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract inflammation	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	20.0% 2/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	15.0% 3/20 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	12.9% 4/31 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Asthma	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 3/59 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Respiratory, thoracic and mediastinal disorders Productive cough	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 3/60 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Acne	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.9% 5/175 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Alopecia	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Blister	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Dermatitis	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Dermatitis diaper	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Dry skin	20.0% 2/10 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Eczema	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.2% 5/120 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.6% 3/117 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/59 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.9% 5/175 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Hyperkeratosis	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Palmar erythema	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	3.2% 1/31 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Pruritus	10.0% 1/10 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	16.7% 1/6 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Rash	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 2/14 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 3/21 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	14.3% 1/7 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 3 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.5% 9/120 • Number of events 11 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 1/60 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.1% 6/117 • Number of events 6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.0% 7/175 • Number of events 10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Rash papular	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	7.1% 1/14 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	4.8% 1/21 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Seborrhoeic dermatitis	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.83% 1/120 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.85% 1/117 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.1% 2/175 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Skin induration	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Skin and subcutaneous tissue disorders Erythema	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	5.0% 1/20 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	9.7% 3/31 • Number of events 5 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	2.5% 3/120 • Number of events 4 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	1.7% 2/117 • Number of events 2 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.57% 1/175 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.
Vascular disorders Hypertension	10.0% 1/10 • Number of events 1 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/6 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/14 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/21 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/7 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/10 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/20 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/31 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/120 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/60 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/117 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/59 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.	0.00% 0/175 • Part 1 and Part 2: Up to approximately 5 years The safety population included all participants who received at least one dose of study medication (risdiplam or placebo) whether prematurely withdrawn or not.

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800 821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Publication restrictions are in place

Restriction type: OTHER