Trial Outcomes & Findings for Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) (NCT NCT00847379)

NCT ID: NCT00847379

Last Updated: 2020-07-15

Results Overview

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function) and severe (interferes significantly with usual function). Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 173 participants
• Primary outcome timeframe: Baseline (Week 48 of Study 007) up to Week 102
• Results posted on: 2020-07-15

Participant Flow

A total of 173 participants were screened for eligibility to enter this extension study after completing the 48-week double-blind study PTC124-GD-007-DMD (NCT00592553). All participants met entry criteria and enrolled in this study.

Participants were categorized by the treatment group to which they had been randomly assigned in Study PTC124-GD-007-DMD (NCT00592553).

Participant milestones

Measure	High-Dose Ataluren/High-Dose Ataluren Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit (Week 48) in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Study STARTED	59	57	57
Overall Study As-treated Population	59	57	57
Overall Study COMPLETED	0	0	0
Overall Study NOT COMPLETED	59	57	57

Reasons for withdrawal

Measure	High-Dose Ataluren/High-Dose Ataluren Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally 3 times a day (TID), 20 milligrams/kilogram (mg/kg) at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit (Week 48) in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Overall Study Study terminated by Sponsor	59	57	57

Baseline Characteristics

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Baseline characteristics by cohort

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Total n=173 Participants Total of all reporting groups
Age, Continuous	9.4 years STANDARD_DEVIATION 2.53 • n=5 Participants	9.7 years STANDARD_DEVIATION 2.89 • n=7 Participants	9.3 years STANDARD_DEVIATION 2.28 • n=5 Participants	9.5 years STANDARD_DEVIATION 2.57 • n=4 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Male	59 Participants n=5 Participants	57 Participants n=7 Participants	57 Participants n=5 Participants	173 Participants n=4 Participants

PRIMARY outcome

Timeframe: Baseline (Week 48 of Study 007) up to Week 102

Population: As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function) and severe (interferes significantly with usual function). Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=173 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Number of Participants With Treatment-Emergent Adverse Events (AEs) Any AEs	52 Participants	49 Participants	48 Participants	149 Participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) Drug-related AEs	10 Participants	10 Participants	22 Participants	42 Participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) Serious AEs	0 Participants	1 Participants	2 Participants	3 Participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) Mild AEs	34 Participants	23 Participants	26 Participants	83 Participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) Moderate AEs	13 Participants	22 Participants	19 Participants	54 Participants
Number of Participants With Treatment-Emergent Adverse Events (AEs) Severe AEs	5 Participants	4 Participants	3 Participants	12 Participants

PRIMARY outcome

Timeframe: Baseline (Week 48 of Study 007) up to Week 102

Population: As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.

Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=173 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Number of Participants With Clinically Significant Abnormal Laboratory Parameters	0 Participants	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

The 6MWD test was performed in a 30 meters long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60 Baseline	351.60 meters Standard Deviation 130.312	375.95 meters Standard Deviation 98.976	356.25 meters Standard Deviation 109.369	361.11 meters Standard Deviation 113.760
Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60 Change at Week 60	-17.23 meters Standard Deviation 39.608	-10.50 meters Standard Deviation 41.390	-7.06 meters Standard Deviation 50.121	-11.56 meters Standard Deviation 43.881

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean activity period/day/visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that greater than (>) 2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was less than (<) 50 percent (%) of the mean active period across all days for that participant's visit.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM) Baseline	759.715 meters Standard Deviation 77.5210	742.223 meters Standard Deviation 107.7264	747.464 meters Standard Deviation 102.6531	750.016 meters Standard Deviation 96.1057
Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM) Change at Week 60	1.946 meters Standard Deviation 61.2247	34.283 meters Standard Deviation 88.1587	-1.866 meters Standard Deviation 93.0531	10.991 meters Standard Deviation 82.6356

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/day/visit during the active periods was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM Baseline	4934.575 steps Standard Deviation 2433.230	4543.766 steps Standard Deviation 2051.300	5175.330 steps Standard Deviation 2307.436	4883.613 steps Standard Deviation 2272.108
Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM Change at Week 60	-357.679 steps Standard Deviation 1232.109	-215.062 steps Standard Deviation 1761.773	-647.504 steps Standard Deviation 1680.522	-409.558 steps Standard Deviation 1566.386

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

The SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). Mean total step count/hour during the active periods for the days in a visit was computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM Baseline	395.491 steps Standard Deviation 200.7671	368.672 steps Standard Deviation 155.6648	420.684 steps Standard Deviation 188.9124	394.787 steps Standard Deviation 183.0994
Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM Change at Week 60	-27.650 steps Standard Deviation 87.8797	-34.693 steps Standard Deviation 128.0609	-54.755 steps Standard Deviation 122.4142	-39.010 steps Standard Deviation 113.3481

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories.

SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear the SAM for at least 9 consecutive days. SAM was used to record the number of strides/minute following each visit. A stride is the leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again (that is, a stride generally equals 2 steps). The maximum continuous 10-minute, 20-minute, 30-minute, and 60-minute total step counts were computed for each participant. For each day, an active period was defined as the first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Baseline: 10-minute total step count	34.603 steps Standard Deviation 12.4886	32.863 steps Standard Deviation 8.8533	35.256 steps Standard Deviation 10.0379	34.241 steps Standard Deviation 10.5911
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Change at Week 60: 10-minute total step count	-1.140 steps Standard Deviation 6.9584	-0.607 steps Standard Deviation 7.2946	-2.372 steps Standard Deviation 7.1735	-1.384 steps Standard Deviation 7.1170
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Baseline: 20-minute total step count	28.138 steps Standard Deviation 11.0629	26.096 steps Standard Deviation 7.9168	28.323 steps Standard Deviation 9.3133	27.526 steps Standard Deviation 9.5426
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Change at Week 60: 20-minute total step count	-1.459 steps Standard Deviation 6.7165	-0.662 steps Standard Deviation 6.2171	-2.510 steps Standard Deviation 6.4346	-1.558 steps Standard Deviation 6.4549
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Baseline: 30-minute total step count	24.222 steps Standard Deviation 9.9576	22.205 steps Standard Deviation 7.1045	24.498 steps Standard Deviation 8.4938	23.648 steps Standard Deviation 8.6306
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Change at Week 60: 30-minute total step count	-1.400 steps Standard Deviation 5.7605	-0.450 steps Standard Deviation 5.6811	-2.457 steps Standard Deviation 5.9579	-1.452 steps Standard Deviation 5.8110
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Baseline: 60-minute total step count	18.019 steps Standard Deviation 7.8145	16.453 steps Standard Deviation 5.6625	18.599 steps Standard Deviation 6.9509	17.691 steps Standard Deviation 6.8991
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM Change at Week 60: 60-minute total step count	-0.977 steps Standard Deviation 3.7328	-0.210 steps Standard Deviation 4.7035	-2.257 steps Standard Deviation 5.1038	-1.162 steps Standard Deviation 4.5803

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories.

SAM is a pedometer (worn on the ankle) that continuously records the number of steps per time interval. Participants were instructed to continue to wear SAM for at least 9 consecutive days. SAM was used to record number of strides/minute following each visit. A stride is leg motion that begins when the foot with SAM leaves the floor and ends when the same foot touches the floor again. Percentage of time during active periods spent at no activity (0 steps/min), low activity (≤15 steps/min), medium activity (16-30 steps/min), and high activity (>30 steps/min) were computed for each participant. For each day, an active period was defined as first time after 3:00 AM that >2 strides/minute were recorded to the last time prior to midnight that >2 strides/minute were recorded. Days were deleted on which such an active period was <50% of the mean active period across all days for that participant's visit.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Baseline: Time spent at no activity	52.818 percentage of time Standard Deviation 14.4129	53.372 percentage of time Standard Deviation 12.2352	50.006 percentage of time Standard Deviation 12.5263	52.094 percentage of time Standard Deviation 13.1188
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Change at Week 60: Time spent at no activity	-0.051 percentage of time Standard Deviation 8.1776	0.830 percentage of time Standard Deviation 11.2481	2.194 percentage of time Standard Deviation 7.8652	0.985 percentage of time Standard Deviation 9.1467
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Baseline: Time spent at low activity	30.677 percentage of time Standard Deviation 7.8531	31.170 percentage of time Standard Deviation 7.2431	32.393 percentage of time Standard Deviation 7.2426	31.392 percentage of time Standard Deviation 7.4462
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Change at Week 60: Time spent at low activity	1.270 percentage of time Standard Deviation 5.8178	1.172 percentage of time Standard Deviation 8.3050	0.252 percentage of time Standard Deviation 5.5885	0.896 percentage of time Standard Deviation 6.6058
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Baseline: Time spent at medium activity	10.248 percentage of time Standard Deviation 4.7620	10.119 percentage of time Standard Deviation 4.4344	10.983 percentage of time Standard Deviation 4.6049	10.442 percentage of time Standard Deviation 4.5901
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Change at Week 60: Time spent at medium activity	-0.542 percentage of time Standard Deviation 2.0960	-1.356 percentage of time Standard Deviation 3.7830	-0.965 percentage of time Standard Deviation 3.3763	-0.944 percentage of time Standard Deviation 3.1392
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Baseline: Time spent at high activity	6.404 percentage of time Standard Deviation 4.5938	5.370 percentage of time Standard Deviation 3.4243	6.673 percentage of time Standard Deviation 4.5847	6.149 percentage of time Standard Deviation 4.2476
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [≤]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60 Change at Week 60: Time spent at high activity	-0.643 percentage of time Standard Deviation 2.2656	-0.520 percentage of time Standard Deviation 2.7196	-1.506 percentage of time Standard Deviation 2.9904	-0.898 percentage of time Standard Deviation 2.6913

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Time to Stand From Supine Position at Week 60 Baseline	13.736 seconds Standard Deviation 11.3353	11.796 seconds Standard Deviation 10.3544	12.398 seconds Standard Deviation 11.6010	12.666 seconds Standard Deviation 11.0678
Change From Baseline in Time to Stand From Supine Position at Week 60 Change at Week 60	0.665 seconds Standard Deviation 3.0080	-0.187 seconds Standard Deviation 4.6289	0.002 seconds Standard Deviation 4.6322	0.166 seconds Standard Deviation 4.1373

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Time to Walk/Run 10 Meters at Week 60 Baseline	8.143 seconds Standard Deviation 5.1475	6.986 seconds Standard Deviation 2.9995	6.967 seconds Standard Deviation 3.5789	7.384 seconds Standard Deviation 4.0530
Change From Baseline in Time to Walk/Run 10 Meters at Week 60 Change at Week 60	1.441 seconds Standard Deviation 4.4822	0.547 seconds Standard Deviation 2.2058	0.758 seconds Standard Deviation 2.6862	0.922 seconds Standard Deviation 3.2905

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Time to Climb 4 Stairs at Week 60 Baseline	9.077 seconds Standard Deviation 9.2543	6.738 seconds Standard Deviation 6.4069	8.071 seconds Standard Deviation 9.2057	7.984 seconds Standard Deviation 8.4076
Change From Baseline in Time to Climb 4 Stairs at Week 60 Change at Week 60	0.831 seconds Standard Deviation 2.4664	0.696 seconds Standard Deviation 4.0707	0.194 seconds Standard Deviation 2.5268	0.574 seconds Standard Deviation 3.0898

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable at specified timepoints.

If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used. Change from baseline data has been reported.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Time to Descend 4 Stairs at Week 60 Baseline	7.685 seconds Standard Deviation 8.9934	5.638 seconds Standard Deviation 5.5635	6.641 seconds Standard Deviation 7.8591	6.675 seconds Standard Deviation 7.6319
Change From Baseline in Time to Descend 4 Stairs at Week 60 Change at Week 60	1.167 seconds Standard Deviation 4.7624	0.626 seconds Standard Deviation 5.2567	-0.648 seconds Standard Deviation 4.9202	0.385 seconds Standard Deviation 5.0045

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and had sufficient baseline and on-treatment data in Study 007e to assess measure of interest, excluding participants who had lost all independent ambulation. 'Number analyzed'=participants evaluable for specified categories.

The heart rate was measured with a Polar RS400 heart rate monitor, which consists of a transmitter strap worn around the chest and a wristwatch receiver. The monitor produces a digital text file with 1 value per minute that represents the mean heart rate for that minute. Mean heart rates values were collected prior to, during, and after the 6MWT. The participant rested for 5 minutes in a sitting position prior to the 6MWT, and the mean heart rate for the last minute of this rest period was collected and documented as the resting heart rate. During the 6MWT, the mean heart rate was collected and documented as the active heart rate. After completing the 6MWT and resting for 3 minutes, the mean heart rate for 1 minute was collected and documented as the recovery heart rate.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=53 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=50 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=49 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=152 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 Change at Week 60: Recovery heart rate	2.1 beats/minute Standard Deviation 12.82	4.6 beats/minute Standard Deviation 12.57	2.3 beats/minute Standard Deviation 11.29	3.0 beats/minute Standard Deviation 12.20
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 Baseline: Resting heart rate	105.3 beats/minute Standard Deviation 13.29	108.8 beats/minute Standard Deviation 10.71	103.7 beats/minute Standard Deviation 12.98	105.9 beats/minute Standard Deviation 12.48
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 Change at Week 60: Resting heart rate	4.0 beats/minute Standard Deviation 13.91	3.7 beats/minute Standard Deviation 10.11	4.8 beats/minute Standard Deviation 10.82	4.2 beats/minute Standard Deviation 11.65
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 Baseline: Active heart rate	138.7 beats/minute Standard Deviation 21.45	144.8 beats/minute Standard Deviation 14.55	138.6 beats/minute Standard Deviation 20.87	140.6 beats/minute Standard Deviation 19.36
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 Change at Week 60: Active heart rate	1.6 beats/minute Standard Deviation 17.65	6.1 beats/minute Standard Deviation 15.45	2.4 beats/minute Standard Deviation 14.87	3.4 beats/minute Standard Deviation 16.07
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400 Baseline: Recovery heart rate	110.0 beats/minute Standard Deviation 13.32	112.0 beats/minute Standard Deviation 12.17	109.4 beats/minute Standard Deviation 13.21	110.4 beats/minute Standard Deviation 12.88

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.

Basic attention and working memory was measured using the digit span task. A series of digits (0-9) were presented to the child in an auditory format only. The task had 2 parts; in the forward condition, the child was requested to repeat back the digits in the order they were presented and in the backward condition, he was requested to reverse the order of presentation. A raw score of the total number of correct responses was converted to an age-scaled-score (z-score) by subtracting the corresponding mean and dividing by the corresponding standard deviation of a reference population for that age.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60 Baseline: Forward condition	4.1 z-score Standard Deviation 2.97	3.6 z-score Standard Deviation 2.54	3.2 z-score Standard Deviation 2.21	3.6 z-score Standard Deviation 2.61
Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60 Change at Week 60: Forward condition	0.0 z-score Standard Deviation 1.53	0.0 z-score Standard Deviation 1.19	0.0 z-score Standard Deviation 1.22	0.0 z-score Standard Deviation 1.32
Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60 Baseline: Backward condition	1.4 z-score Standard Deviation 0.98	1.3 z-score Standard Deviation 0.82	1.2 z-score Standard Deviation 0.66	1.3 z-score Standard Deviation 0.84
Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60 Change at Week 60: Backward condition	0.1 z-score Standard Deviation 0.86	-0.1 z-score Standard Deviation 0.66	0.0 z-score Standard Deviation 0.58	0.0 z-score Standard Deviation 0.71

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.

HRQL was measured via the PedsQL. The generic core module (including physical, emotional, social and school functioning scales) comprises 23 questions and the fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. The PedsQL was completed by both the participant and/or a parent/caregiver. Examples of items in each of the generic core module scales include: "It is hard for me to run"; "I feel sad or blue"; "I cannot do things that other kids my age can do;" and "It is hard to pay attention in class." Each of the generic core module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating better health-related quality of life. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 60.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: Physical functioning score	63.8145 units on a scale Standard Deviation 24.46918	63.3019 units on a scale Standard Deviation 22.72039	60.4865 units on a scale Standard Deviation 22.33484	62.5995 units on a scale Standard Deviation 23.11654
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: Physical functioning score	-0.5982 units on a scale Standard Deviation 14.91137	-1.2538 units on a scale Standard Deviation 14.60384	1.2755 units on a scale Standard Deviation 19.01169	-0.1804 units on a scale Standard Deviation 16.23570
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: Emotional functioning score	76.852 units on a scale Standard Deviation 21.1530	73.241 units on a scale Standard Deviation 20.1681	73.265 units on a scale Standard Deviation 22.2568	74.490 units on a scale Standard Deviation 21.1086
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: Emotional functioning score	-1.675 units on a scale Standard Deviation 13.6361	2.128 units on a scale Standard Deviation 14.9181	-0.995 units on a scale Standard Deviation 13.1621	-0.223 units on a scale Standard Deviation 13.9106
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: Social functioning score	72.5000 units on a scale Standard Deviation 21.60429	68.6111 units on a scale Standard Deviation 21.37748	71.3000 units on a scale Standard Deviation 21.32858	70.7911 units on a scale Standard Deviation 21.36670
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: Social functioning score	-2.2000 units on a scale Standard Deviation 13.48317	0.8511 units on a scale Standard Deviation 17.36185	0.3500 units on a scale Standard Deviation 15.15237	-0.3571 units on a scale Standard Deviation 15.32468
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: School functioning score	72.019 units on a scale Standard Deviation 20.7756	71.111 units on a scale Standard Deviation 18.2918	69.063 units on a scale Standard Deviation 20.3861	70.779 units on a scale Standard Deviation 19.7213
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: School functioning score	-1.064 units on a scale Standard Deviation 16.5153	-1.630 units on a scale Standard Deviation 17.7029	1.809 units on a scale Standard Deviation 16.9214	-0.286 units on a scale Standard Deviation 16.9931

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.

HRQL was measured via the PedsQL. The generic core module (including physical, emotional, social and school functioning scales) comprises 23 questions and the fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. The PedsQL was completed by both the participant and/or a parent/caregiver. Examples of items in each of the generic core module scales include: "It is hard for me to run"; "I feel sad or blue"; "I cannot do things that other kids my age can do;" and "It is hard to pay attention in class." Each of the generic core module items was scored on a 5-point likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating better health-related quality of life. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 48.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: Physical functioning score	56.8217 units on a scale Standard Deviation 23.80718	52.5162 units on a scale Standard Deviation 20.02845	52.6307 units on a scale Standard Deviation 24.35727	54.0152 units on a scale Standard Deviation 22.78661
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: Physical functioning score	-0.8499 units on a scale Standard Deviation 12.75478	-1.1662 units on a scale Standard Deviation 13.27447	-0.1736 units on a scale Standard Deviation 14.75049	-0.7143 units on a scale Standard Deviation 13.55924
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: Emotional functioning score	74.7368 units on a scale Standard Deviation 17.73870	72.5455 units on a scale Standard Deviation 16.04025	68.2813 units on a scale Standard Deviation 22.05309	71.8676 units on a scale Standard Deviation 18.87211
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: Emotional functioning score	-1.9231 units on a scale Standard Deviation 11.11289	-0.3061 units on a scale Standard Deviation 13.32323	-1.9213 units on a scale Standard Deviation 16.86642	-1.4113 units on a scale Standard Deviation 13.95342
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: Social functioning score	61.9518 units on a scale Standard Deviation 19.07606	61.6136 units on a scale Standard Deviation 15.44605	59.7545 units on a scale Standard Deviation 21.34593	61.1086 units on a scale Standard Deviation 18.71300
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: Social functioning score	1.5144 units on a scale Standard Deviation 13.03356	-0.7908 units on a scale Standard Deviation 11.34994	0.3472 units on a scale Standard Deviation 12.76238	0.3790 units on a scale Standard Deviation 12.38287
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Baseline: School functioning score	68.6607 units on a scale Standard Deviation 16.05363	64.0000 units on a scale Standard Deviation 15.07389	65.9091 units on a scale Standard Deviation 18.20894	66.2048 units on a scale Standard Deviation 16.50694
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60 Change at Week 60: School functioning score	0.8000 units on a scale Standard Deviation 9.60230	1.6667 units on a scale Standard Deviation 10.93177	-1.0377 units on a scale Standard Deviation 12.60946	0.4305 units on a scale Standard Deviation 11.13464

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable at specified timepoints.

HRQL was measured via the PedsQL. The fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. PedsQL was completed by both the participant and/or a parent/caregiver. Fatigue-specific module obtains information relating to items such as: "I feel too tired to do things that I like to do"; "I spend a lot of time in bed"; and "I have trouble remembering more than one thing at a time;" Each of the fatigue-specific module items was scored on a 5-point Likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating less fatigue. Total score was the sum of all items over the number of items answered on all scales. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 60.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Participant-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60 Baseline	76.7968 units on a scale Standard Deviation 18.31310	72.7833 units on a scale Standard Deviation 20.95237	72.5947 units on a scale Standard Deviation 17.32956	74.1111 units on a scale Standard Deviation 18.92041
Change From Baseline in Participant-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60 Change at Week 60	-2.0484 units on a scale Standard Deviation 9.77928	0.3650 units on a scale Standard Deviation 15.94374	-1.8049 units on a scale Standard Deviation 11.12724	-1.2005 units on a scale Standard Deviation 12.42615

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable at specified timepoints.

HRQL was measured via the PedsQL. The fatigue-specific module (including general fatigue, sleep/rest fatigue, and cognitive fatigue scales) comprises an additional 18 questions. The PedsQL was completed by both the participant and/or a parent/caregiver. Fatigue-specific module obtains information relating to items such as: "I feel too tired to do things that I like to do"; "I spend a lot of time in bed"; and "I have trouble remembering more than one thing at a time;" Each of the fatigue-specific module items was scored on a 5-point likert response scale from 0 (never a problem) to 4 (almost always a problem). Scores were transformed on a scale from 0 to 100 (0=100, 1=75, 2=50, 3=25, 4=0), with higher scores indicating less fatigue. Total score was the sum of all items over the number of items answered on all scales. Change from Baseline was calculated by subtracting the Baseline value from the value at Week 60.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Parent/Caregiver-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60 Baseline	75.7864 units on a scale Standard Deviation 13.88014	71.6596 units on a scale Standard Deviation 12.78488	71.1062 units on a scale Standard Deviation 13.62739	72.8724 units on a scale Standard Deviation 13.53355
Change From Baseline in Parent/Caregiver-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60 Change at Week 60	-0.3272 units on a scale Standard Deviation 6.64218	1.2219 units on a scale Standard Deviation 10.11693	0.2269 units on a scale Standard Deviation 8.55130	0.3341 units on a scale Standard Deviation 8.44319

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Non-Ambulatory 007e population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, and who had lost all independent ambulation prior to entering Study 007e. 'Number analyzed'=participants evaluable at specified timepoints.

Activities of daily living were measured using the EK scale in all participants who were unable to complete the 6MWT at Screening/Baseline on Day 1. The EK scale is an ordinal scale ranging from 0 to 30 points where 0 represents the highest level of independent function and 30 the lowest. The scale consists of 10 categories (each scored 0 to 3), involving different functional domains including 1) ability to use wheelchair, 2) ability to transfer from wheelchair, 3) ability to stand, 4) ability to balance in the wheelchair, 5) ability to move the arms, 6) ability to use the hands and arms when eating, 7) ability to turn in bed, 8) ability to cough, 9) ability to speak, and 10) physical well-being. The administration of the EK scale consisted of an interview of the participant to capture how he performed the tasks of daily life (as described by Categories 1 to 9) and how he perceived his well-being (as described by Category 10).

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=5 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=4 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=6 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=15 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Participant and Parent/Caregiver Reported Activities of Daily Living of Participants Who Were Unable to Complete the 6MWT (Nonambulatory Participants), as Measured by the Egen Klassifikation (EK) Scale at Week 60 Baseline	3.0 units on a scale Standard Deviation 2.00	4.3 units on a scale Standard Deviation 0.50	3.2 units on a scale Standard Deviation 1.92	3.5 units on a scale Standard Deviation 1.61
Change From Baseline in Participant and Parent/Caregiver Reported Activities of Daily Living of Participants Who Were Unable to Complete the 6MWT (Nonambulatory Participants), as Measured by the Egen Klassifikation (EK) Scale at Week 60 Change at Week 60	1.5 units on a scale Standard Deviation 0.58	0.8 units on a scale Standard Deviation 1.26	-0.8 units on a scale Standard Deviation 1.48	0.4 units on a scale Standard Deviation 1.50

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable for specified categories.

TSQM consisted of 14 questions about treatment satisfaction with drug in 4 domains: Effectiveness (Questions 1-3 scored as 1 [extremely dissatisfied] to 7 [extremely satisfied]), Side Effects (question 4 scored as 0 [no] or 1 [yes]; question 5 scored as 1 [extremely bothersome] to 5 [not at all bothersome]; questions 6 - 8 scored as 1 [a great deal] to 5 [not at all]), Convenience (questions 9 and 10 scored as 1 [extremely difficult] to 7 [extremely easy]; question 11 scored as 1 [extremely inconvenient] to 5 [extremely convenient]) and Global Satisfaction (question 12 scored as 1 [not at all confident] to 7 [extremely confident]; question 13 scored as 1 [not at all certain] to 5 [extremely certain]; question 14 scored as 1 [extremely dissatisfied] to 5 [extremely satisfied]). The scores of each of the domains were added together and an algorithm was used to create a score of 0 to 100, with higher scores indicating better treatment satisfaction.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Baseline: Effectiveness score	56.8134 units on a scale Standard Deviation 26.72451	53.8580 units on a scale Standard Deviation 21.53292	50.4274 units on a scale Standard Deviation 22.96804	53.7212 units on a scale Standard Deviation 23.82541
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Change at Week 60: Effectiveness score	2.7778 units on a scale Standard Deviation 15.52191	5.6159 units on a scale Standard Deviation 19.53233	1.7778 units on a scale Standard Deviation 14.99307	3.3295 units on a scale Standard Deviation 16.68460
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Baseline: Side-effects score	96.8182 units on a scale Standard Deviation 10.81681	97.7273 units on a scale Standard Deviation 7.64176	96.8364 units on a scale Standard Deviation 8.94695	97.1291 units on a scale Standard Deviation 9.18246
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Change at Week 60: Side-effects score	0.7353 units on a scale Standard Deviation 12.16084	-1.0638 units on a scale Standard Deviation 7.74500	-1.2587 units on a scale Standard Deviation 10.84441	-0.4994 units on a scale Standard Deviation 10.43907
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Baseline: Convenience score	57.4074 units on a scale Standard Deviation 16.10609	57.9798 units on a scale Standard Deviation 19.05889	60.5967 units on a scale Standard Deviation 16.65858	58.6345 units on a scale Standard Deviation 17.26216
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Change at Week 60: Convenience score	0.8386 units on a scale Standard Deviation 12.39372	-1.6548 units on a scale Standard Deviation 11.75175	1.3333 units on a scale Standard Deviation 9.80472	0.2222 units on a scale Standard Deviation 11.38059
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Baseline: Global satisfaction score	61.4286 units on a scale Standard Deviation 25.95857	60.4870 units on a scale Standard Deviation 23.30835	56.7791 units on a scale Standard Deviation 21.25768	59.5819 units on a scale Standard Deviation 23.54109
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60 Change at Week 60: Global satisfaction score	-0.2695 units on a scale Standard Deviation 13.57882	1.2538 units on a scale Standard Deviation 15.30213	-0.4643 units on a scale Standard Deviation 14.78188	0.1429 units on a scale Standard Deviation 14.45922

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007), Week 60

Population: Evaluable population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e, who had sufficient baseline and on-treatment data in Study 007e to assess the measure of interest. 'Number analyzed'=participants evaluable at specified timepoints.

Blood samples collected for chemistry assays were used to quantify serum CK concentrations. Serum CK was assessed as a potential biomarker for muscle fragility, with a reduction in serum CK considered to be a positive outcome.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=58 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=56 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=56 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=170 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 60 Baseline	9163.1 units/liter (U/L) Standard Deviation 5542.80	10009.1 units/liter (U/L) Standard Deviation 5828.26	9150.7 units/liter (U/L) Standard Deviation 5657.67	9442.8 units/liter (U/L) Standard Deviation 5656.99
Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 60 Change at Week 60	-455.1 units/liter (U/L) Standard Deviation 4962.00	-520.5 units/liter (U/L) Standard Deviation 6164.96	-334.3 units/liter (U/L) Standard Deviation 4810.45	-435.1 units/liter (U/L) Standard Deviation 5310.37

SECONDARY outcome

Timeframe: Baseline (Week 48 of Study 007) to Week 96

Population: As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.

Study drug compliance was assessed by participant daily diary and quantification of used and unused study drug. Compliance was assessed in terms of the percentage of drug actually taken relative to the amount that should have been taken during the study.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=173 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Study Drug Compliance	0.00 percentage of drug Interval 0.0 to 45.1	0.00 percentage of drug Interval 0.0 to 10.5	0.00 percentage of drug Interval 0.0 to 36.1	0.00 percentage of drug Interval 0.0 to 45.1

SECONDARY outcome

Timeframe: Pre-morning dose (0 hour) at Baseline (Week 48 of 007 study), Weeks 54, 60, 72, 84, and 96

Population: As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e. 'Number analyzed'=participants evaluable at specified timepoints.

Plasma samples for the determination of ataluren concentrations were analyzed at the bioanalytical laboratory for ataluren parent drug using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation (LLOQ) of 0.5 micrograms/millilitre (mcg/mL). Values below the LLOQ were set to 0.

Outcome measures

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 Participants Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 Participants Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 Participants Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=173 Participants All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Trough Ataluren Plasma Concentration Week 48	12.656 mcg/mL Standard Deviation 10.8951	4.541 mcg/mL Standard Deviation 3.4381	0.000 mcg/mL Standard Deviation 0.0000	5.773 mcg/mL Standard Deviation 8.4333
Trough Ataluren Plasma Concentration Week 54	10.801 mcg/mL Standard Deviation 8.4858	13.941 mcg/mL Standard Deviation 14.5222	11.564 mcg/mL Standard Deviation 9.3312	12.097 mcg/mL Standard Deviation 11.1172
Trough Ataluren Plasma Concentration Week 60	11.547 mcg/mL Standard Deviation 9.6034	13.830 mcg/mL Standard Deviation 11.9096	13.302 mcg/mL Standard Deviation 9.4765	12.890 mcg/mL Standard Deviation 10.3574
Trough Ataluren Plasma Concentration Week 72	12.279 mcg/mL Standard Deviation 10.5894	13.930 mcg/mL Standard Deviation 10.6393	10.948 mcg/mL Standard Deviation 6.3465	12.424 mcg/mL Standard Deviation 9.4948
Trough Ataluren Plasma Concentration Week 84	15.232 mcg/mL Standard Deviation 8.8909	14.773 mcg/mL Standard Deviation 10.4404	11.082 mcg/mL Standard Deviation 14.6331	13.901 mcg/mL Standard Deviation 10.7415
Trough Ataluren Plasma Concentration Week 96	8.430 mcg/mL	5.685 mcg/mL Standard Deviation 2.7648	—	6.600 mcg/mL Standard Deviation 2.5167

Adverse Events

High-Dose Ataluren/High-Dose Ataluren

Serious events: 0 serious events

Other events: 53 other events

Deaths: 0 deaths

Low-Dose Ataluren/High-Dose Ataluren

Serious events: 1 serious events

Other events: 51 other events

Deaths: 0 deaths

Placebo/High-Dose Ataluren

Serious events: 2 serious events

Other events: 51 other events

Deaths: 0 deaths

Overall Participants: High-Dose Ataluren

Serious events: 3 serious events

Other events: 155 other events

Deaths: 0 deaths

Serious adverse events

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 participants at risk Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 participants at risk Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 participants at risk Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=173 participants at risk All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Injury, poisoning and procedural complications Femur fracture	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Vascular disorders Hypertension	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.

Other adverse events

Measure	High-Dose Ataluren/High-Dose Ataluren n=59 participants at risk Participants who were randomized to receive high-dose ataluren in study PTC124-GD-007-DMD, continued to receive ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Low-Dose Ataluren/High-Dose Ataluren n=57 participants at risk Participants who were randomized to receive low-dose ataluren in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Placebo/High-Dose Ataluren n=57 participants at risk Participants who were randomized to receive placebo in study PTC124-GD-007-DMD, received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of placebo at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.	Overall Participants: High-Dose Ataluren n=173 participants at risk All participants received ataluren suspension orally TID, 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, was initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose was increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level was well tolerated.
Ear and labyrinth disorders Deafness	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Ear and labyrinth disorders Ear pain	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Blood and lymphatic system disorders Lymphadenopathy	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Blood and lymphatic system disorders Anaemia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Blood and lymphatic system disorders Microcytic anaemia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Blood and lymphatic system disorders Neutropenia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Cardiac disorders Dilatation ventricular	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Cardiac disorders Arrhythmia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Cardiac disorders Cardiomyopathy	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Cardiac disorders Left ventricular hypertrophy	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Cardiac disorders Tachycardia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Congenital, familial and genetic disorders Kidney malformation	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Ear and labyrinth disorders Tympanic membrane hyperaemia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Ear and labyrinth disorders Vertigo	1.7% 1/59 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Endocrine disorders Cushingoid	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Cataract	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Myopia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Abnormal sensation in eye	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Conjunctivitis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Hypermetropia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Photophobia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Strabismus	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Eye disorders Vision blurred	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Vomiting	18.6% 11/59 • Number of events 16 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	21.1% 12/57 • Number of events 23 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	35.1% 20/57 • Number of events 33 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	24.9% 43/173 • Number of events 72 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Diarrhoea	5.1% 3/59 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	19.3% 11/57 • Number of events 13 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	10.5% 6/57 • Number of events 8 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	11.6% 20/173 • Number of events 25 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Abdominal pain	10.2% 6/59 • Number of events 10 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	10.5% 6/57 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	9.2% 16/173 • Number of events 21 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Abdominal pain upper	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	15.8% 9/57 • Number of events 11 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.7% 15/173 • Number of events 18 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Flatulence	11.9% 7/59 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.1% 14/173 • Number of events 14 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Nausea	8.5% 5/59 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	6.4% 11/173 • Number of events 12 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Dyspepsia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Constipation	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Stomach discomfort	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Aerophagia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Gastrooesophageal reflux disease	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Abdominal discomfort	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Abdominal pain lower	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Abdominal tenderness	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Abnormal faeces	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Aphthous stomatitis	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Chapped lips	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Duodenogastric reflux	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Eructation	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Faecal incontinence	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Gastritis	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Gastrointestinal sounds abnormal	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Gingivitis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Oral pain	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Gastrointestinal disorders Tongue disorder	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Disease progression	15.3% 9/59 • Number of events 9 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	12.3% 7/57 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	11.0% 19/173 • Number of events 19 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Asthenia	3.4% 2/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.8% 5/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.2% 9/173 • Number of events 10 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Gait disturbance	6.8% 4/59 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.2% 9/173 • Number of events 9 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Pyrexia	6.8% 4/59 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.2% 9/173 • Number of events 11 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Fatigue	5.1% 3/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Malaise	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
General disorders Oedema peripheral	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Immune system disorders Hypersensitivity	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Influenza	11.9% 7/59 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.8% 5/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	10.5% 6/57 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	10.4% 18/173 • Number of events 18 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Nasopharyngitis	5.1% 3/59 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	14.0% 8/57 • Number of events 10 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.8% 5/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	9.2% 16/173 • Number of events 19 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Upper respiratory tract infection	10.2% 6/59 • Number of events 8 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.8% 5/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.1% 14/173 • Number of events 17 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Ear infection	3.4% 2/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	4.6% 8/173 • Number of events 10 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Gastroenteritis viral	5.1% 3/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	4.0% 7/173 • Number of events 8 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Gastroenteritis	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Otitis media	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Rhinitis	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Bronchitis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Croup infectious	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Lower respiratory tract infection	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Paronychia	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Sinusitis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Urinary tract infection	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Viral infection	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Enterobiasis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Gastrointestinal infection	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Molluscum contagiosum	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Pharyngitis	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Pharyngitis streptococcal	3.4% 2/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Tinea pedis	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Conjunctivitis infective	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Helicobacter infection	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Localised infection	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Lung infection	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Onychomycosis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Oral herpes	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Otitis externa	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Pneumonia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Infections and infestations Scarlet fever	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Fall	8.5% 5/59 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.8% 5/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	8.1% 14/173 • Number of events 15 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Contusion	3.4% 2/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Iliotibial band syndrome	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Joint sprain	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Arthropod bite	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Femur fracture	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Limb injury	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Lower limb fracture	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Arthropod sting	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Excoriation	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Facial bones fracture	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Foot fracture	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Head injury	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Incision site erythema	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Lumbar vertebral fracture	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Mouth injury	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Muscle strain	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Overdose	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Procedural pain	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Skin laceration	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Spinal compression fracture	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Wound	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Injury, poisoning and procedural complications Wound dehiscence	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Investigations Blood sodium increased	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Investigations Weight decreased	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Investigations Blood bicarbonate decreased	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Investigations Blood triglycerides increased	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Investigations Breath sounds abnormal	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Investigations Weight increased	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Metabolism and nutrition disorders Decreased appetite	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Metabolism and nutrition disorders Dehydration	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Metabolism and nutrition disorders Hypovitaminosis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Metabolism and nutrition disorders Insulin resistance	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Metabolism and nutrition disorders Iron deficiency	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Back pain	6.8% 4/59 • Number of events 11 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	6.4% 11/173 • Number of events 21 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Muscular weakness	8.5% 5/59 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	6.4% 11/173 • Number of events 12 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Pain in extremity	8.5% 5/59 • Number of events 8 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.8% 10/173 • Number of events 14 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Joint contracture	6.8% 4/59 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 8 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.2% 9/173 • Number of events 13 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Arthralgia	5.1% 3/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	4.0% 7/173 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Muscle tightness	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 6/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Lordosis	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Myalgia	3.4% 2/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Muscle spasms	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Osteoporosis	5.1% 3/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Tendinous contracture	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Muscle atrophy	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Scoliosis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Tendon disorder	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Coccydynia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Groin pain	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Growth retardation	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Joint instability	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Muscle contracture	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Musculoskeletal pain	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Neck pain	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Musculoskeletal and connective tissue disorders Musculoskeletal stiffness	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin papilloma	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.0% 4/57 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 6/173 • Number of events 7 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Melanocytic naevus	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Headache	8.5% 5/59 • Number of events 48 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	26.3% 15/57 • Number of events 48 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	24.6% 14/57 • Number of events 17 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	19.7% 34/173 • Number of events 113 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Migraine	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Areflexia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Dizziness	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Hypertonia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Hypotonia	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Memory impairment	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Partial seizures	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Sedation	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Nervous system disorders Sinus headache	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Attention deficit/hyperactivity disorder	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.9% 5/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Abnormal behaviour	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Aggression	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Obsessive-compulsive disorder	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Tic	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Depression	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Frustration	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Psychiatric disorders Oppositional defiant disorder	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Enuresis	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 6/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Urine abnormality	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Pollakiuria	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Urinary incontinence	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Dysuria	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Haematuria	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Kidney enlargement	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Micturition urgency	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Pyelocaliectasis	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Renal cyst	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Renal and urinary disorders Ureteric dilatation	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Vascular disorders Hot flush	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Vascular disorders Hypertension	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 4 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Rash	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	5.3% 3/57 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 6/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Dry skin	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.7% 3/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Ecchymosis	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Eczema	1.7% 1/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Pruritus	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Acne	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Cold sweat	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Hyperkeratosis	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Keratosis pilaris	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Petechiae	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Pityriasis rosea	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Rash maculo-papular	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Rash papular	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Skin chapped	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Skin depigmentation	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Skin discolouration	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Skin exfoliation	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Skin odour abnormal	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Skin and subcutaneous tissue disorders Swelling face	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Cough	10.2% 6/59 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	10.5% 6/57 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	7.5% 13/173 • Number of events 13 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	6.8% 4/59 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	4.0% 7/173 • Number of events 9 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Nasal congestion	3.4% 2/59 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 2/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	3.5% 6/173 • Number of events 6 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Epistaxis	5.1% 3/59 • Number of events 3 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	2.3% 4/173 • Number of events 5 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Wheezing	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.2% 2/173 • Number of events 2 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Asthma	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Pharyngeal erythema	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Postnasal drip	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Productive cough	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Respiratory tract congestion	1.7% 1/59 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Sinus congestion	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.
Respiratory, thoracic and mediastinal disorders Snoring	0.00% 0/59 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.00% 0/57 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	1.8% 1/57 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.	0.58% 1/173 • Number of events 1 • Baseline (Week 48 of Study 007) up to Week 102 As-treated population included all participants who completed Study 007 and received ≥1 dose of ataluren in Study 007e.

Additional Information

Patient Advocacy

PTC Therapeutics, Inc.

Phone: 1-866-562-4620

Email: medinfo@ptcbio.com

Results disclosure agreements

• Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
• Publication restrictions are in place

Restriction type: OTHER