Trial Outcomes & Findings for A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy (NCT NCT03760029)

Last Updated: 2024-09-19

Results Overview

Participant's age at life-altering clinical milestones- failure to walk was calculated based on the birthdate and the date of failure to walk as reported by caregiver during 30 months of this study. Participants who were not reported being failure to walk by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

Recruitment status

COMPLETED

Study phase

Target enrollment

312 participants

Primary outcome timeframe

Up to Month 30

Results posted on

2024-09-19

Participant Flow

This was a multicenter, prospective study to describe natural history of Duchenne muscular dystrophy (DMD) in Chinese male participants.

A total of 318 participants were screened of which 6 were screen failures and 312 participants were enrolled at 6 sites in China.

Participant milestones

Participant milestones
Measure	Ambulatory Participants With Age <6 Years Participants diagnosed with DMD who were ambulant and were less than 6 years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the standard of care (SOC).	Ambulatory Participants With Age >=6 Years Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.
Overall Study STARTED	99	177	36
Overall Study COMPLETED	91	166	33
Overall Study NOT COMPLETED	8	11	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Ambulatory Participants With Age <6 Years Participants diagnosed with DMD who were ambulant and were less than 6 years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the standard of care (SOC).	Ambulatory Participants With Age >=6 Years Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.
Overall Study Death	1	0	0
Overall Study Lost to Follow-up	0	2	1
Overall Study Withdrawal by Subject	1	4	1
Overall Study Withdrawal by parent/guardian	4	4	1
Overall Study Other	2	1	0

Baseline Characteristics

A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ambulatory Participants With Age <6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were less than 6 years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the standard of care (SOC).	Ambulatory Participants With Age >=6 Years n=177 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total n=312 Participants Total of all reporting groups
Age, Continuous	4.0 Years STANDARD_DEVIATION 1.23 • n=5 Participants	9.0 Years STANDARD_DEVIATION 1.99 • n=7 Participants	12.7 Years STANDARD_DEVIATION 2.71 • n=5 Participants	7.9 Years STANDARD_DEVIATION 3.43 • 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	99 Participants n=5 Participants	177 Participants n=7 Participants	36 Participants n=5 Participants	312 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	99 Participants n=5 Participants	177 Participants n=7 Participants	36 Participants n=5 Participants	312 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	99 Participants n=5 Participants	177 Participants n=7 Participants	36 Participants n=5 Participants	312 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants

PRIMARY outcome

Timeframe: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, 'Number of Participants Analyzed' signifies participants included in the Kaplan-Meier analysis. No participant in "Ambulatory participants aged \< 6 years" had an event of "failure to walk".

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Age of Participants When They Failed to Walk	14.4 Years Interval 13.9 to The upper limit of the 95% confidence interval was not reached due to less number of participants with an event.	NA Years Data could not be estimated as no participants had "failure to walk" event	14.4 Years Interval 13.9 to The upper limit of the 95% confidence interval was not reached due to less number of participants with an event.	11.1 Years Interval 10.0 to 11.4	13.1 Years Interval 12.5 to 14.1

PRIMARY outcome

Timeframe: Up to Month 30

Participant's age at life-altering clinical milestones- failure to stand was calculated based on the birthdate and the date of failure to stand as reported by caregiver during 30 months of this study. Participants who were not reported being failure to stand by their caregivers were censored on the day of their last visit. Kaplan-Meier method was used for analysis.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Age of Participants When They Failed to Stand	14.4 Years Interval 13.9 to The upper limit of the 95% confidence interval was not reached due to less number of participants with an event.	NA Years Data could not be estimated as no participants had "failure to stand" event	14.4 Years Interval 13.9 to The upper limit of the 95% confidence interval was not reached due to less number of participants with an event.	11.0 Years Interval 10.5 to 11.5	13.1 Years Interval 12.9 to 14.4

PRIMARY outcome

Timeframe: Up to Month 30

Participant's age at life-altering clinical milestones- failure to self-feed during 30 months of this study was analyzed using the Kaplan-Meier method. Age was summarized in years.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Age of Participants When They Failed to Self-feed	NA Years Data could not be estimated as no participants had "failure to self-feed" event	NA Years Data could not be estimated as no participants had "failure to self-feed" event	NA Years Data could not be estimated as no participants had "failure to self-feed" event	20.0 Years Interval 17.0 to The upper limit of the 95% confidence interval was not reached due to less number of participants with an event.	20.0 Years Interval 17.0 to The upper limit of the 95% confidence interval was not reached due to less number of participants with an event.

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (example \[e.g.\] standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=218 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=64 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=154 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Northstar Ambulatory Assessment (NSAA) Total Score at Month 6: Ambulatory Participants Aged >=3 Years	-0.3 Units on a scale Standard Deviation 4.24	1.3 Units on a scale Standard Deviation 4.44	-1.0 Units on a scale Standard Deviation 3.98	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=196 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=51 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=145 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in NSAA Total Score at Month 12: Ambulatory Participants Aged >=3 Years	-0.9 Units on a scale Standard Deviation 5.42	2.7 Units on a scale Standard Deviation 5.37	-2.1 Units on a scale Standard Deviation 4.88	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=150 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=47 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=103 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in NSAA Total Score at Month 18: Ambulatory Participants Aged >=3 Years	-1.2 Units on a scale Standard Deviation 5.93	4.1 Units on a scale Standard Deviation 5.12	-3.6 Units on a scale Standard Deviation 4.53	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=167 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=53 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=114 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in NSAA Total Score at Month 24: Ambulatory Participants Aged >=3 Years	-2.1 Units on a scale Standard Deviation 6.50	3.9 Units on a scale Standard Deviation 4.67	-4.9 Units on a scale Standard Deviation 5.22	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

NSAA is a 17-item test that grades performance of various functional skills using the following scale: 0 (unable to achieve goal independently), 1 (modified method but achieves goal with no physical assistance), or 2 ("normal"- no obvious modification of activity). The scale assesses activities required to remain functionally ambulant (e.g. rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA total score was calculated by adding the responses of all 17 items and ranged from 0 to 34, with higher scores indicating better function. NSAA was only performed in ambulatory participants aged \>=3 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=85 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=66 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in NSAA Total Score at Month 30: Ambulatory Participants Aged >=3 Years	-3.6 Units on a scale Standard Deviation 6.84	4.1 Units on a scale Standard Deviation 5.09	-5.9 Units on a scale Standard Deviation 5.56	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

PUL 2.0 scale is a 22-item scale used to assess the change that occurs in motor performance of the upper limb overtime from when a participant is still ambulant to the time participant loses all arm function when non-ambulant. PUL 2.0 includes an entry item to define broad starting functional level and 22 items subdivided into shoulder level (six items), mid-level (nine items), and distal level (seven items). Each dimension (shoulder, mid, distal) can be scored separately. There is maximum score of 12 for shoulder level, 17 for mid-level, and 13 for distal level. The total score was calculated by adding three level scores and ranged from 0-42. Higher score indicates better upper limb function. PUL 2.0 total score was assessed in participants aged \>=10 years only as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=50 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=46 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Performance of Upper Limb (PUL) 2.0 Total Score at Month 6: Participants Aged >=10 Years	-0.1 Units on a scale Standard Deviation 2.31	-0.0 Units on a scale Standard Deviation 2.33	-0.8 Units on a scale Standard Deviation 2.22	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=66 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=43 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in PUL 2.0 Total Score at Month 12: Participants Aged >=10 Years	-0.7 Units on a scale Standard Deviation 2.22	-0.6 Units on a scale Standard Deviation 2.16	-0.9 Units on a scale Standard Deviation 2.37	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=31 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=27 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in PUL 2.0 Total Score at Month 18: Participants Aged >=10 Years	-2.1 Units on a scale Standard Deviation 3.23	-1.7 Units on a scale Standard Deviation 3.22	-4.3 Units on a scale Standard Deviation 2.75	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=56 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=35 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=21 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in PUL 2.0 Total Score at Month 24: Participants Aged >=10 Years	-2.8 Units on a scale Standard Deviation 3.65	-2.8 Units on a scale Standard Deviation 3.92	-2.9 Units on a scale Standard Deviation 3.23	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=22 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=18 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in PUL 2.0 Total Score at Month 30: Participants Aged >=10 Years	-2.1 Units on a scale Standard Deviation 3.21	-1.7 Units on a scale Standard Deviation 3.37	-3.8 Units on a scale Standard Deviation 1.71	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The rise from floor velocity was defined as the reciprocal of the time (in seconds) to rise from floor. The rise from floor test was performed only in ambulatory participants aged \>=3 years old as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=230 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=155 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Rise From Floor Velocity at Month 6: Ambulatory Participants Aged >=3 Years Only	0.0 Rise per second Standard Deviation 0.06	0.0 Rise per second Standard Deviation 0.07	-0.0 Rise per second Standard Deviation 0.05	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=205 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=60 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=145 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Rise From Floor Velocity at Month 12: Ambulatory Participants Aged >=3 Years Only	-0.0 Rise per second Standard Deviation 0.08	0.0 Rise per second Standard Deviation 0.08	-0.0 Rise per second Standard Deviation 0.07	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=161 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=57 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=104 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Rise From Floor Velocity at Month 18: Ambulatory Participants Aged >=3 Years Only	-0.0 Rise per second Standard Deviation 0.08	0.0 Rise per second Standard Deviation 0.08	-0.0 Rise per second Standard Deviation 0.06	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=176 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=61 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=115 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Rise From Floor Velocity at Month 24: Ambulatory Participants Aged >=3 Years Only	-0.0 Rise per second Standard Deviation 0.09	0.0 Rise per second Standard Deviation 0.09	-0.1 Rise per second Standard Deviation 0.07	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=89 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=22 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=67 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Rise From Floor Velocity at Month 30: Ambulatory Participants Aged >=3 Years Only	-0.1 Rise per second Standard Deviation 0.10	0.0 Rise per second Standard Deviation 0.10	-0.1 Rise per second Standard Deviation 0.08	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The 10 meter walk or run test was performed as part of NSAA. The 10 meter walk or run velocity was defined as the reciprocal of the time (in seconds) to complete the 10 meter run or walk test. The 10 meter walk or run test was performed in ambulatory children \>=3 years old only as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=227 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=152 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 6: Ambulatory Participants Aged >=3 Years	0.0 Meters per second Standard Deviation 0.35	0.2 Meters per second Standard Deviation 0.42	-0.1 Meters per second Standard Deviation 0.27	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=200 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=60 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=140 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 12: Ambulatory Participants Aged >=3 Years	-0.0 Meters per second Standard Deviation 0.40	0.3 Meters per second Standard Deviation 0.35	-0.1 Meters per second Standard Deviation 0.34	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=159 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=57 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=102 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 18: Ambulatory Participants Aged >=3 Years	-0.0 Meters per second Standard Deviation 0.50	0.4 Meters per second Standard Deviation 0.41	-0.3 Meters per second Standard Deviation 0.34	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=171 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=62 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=109 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 24: Ambulatory Participants Aged >=3 Years	-0.1 Meters per second Standard Deviation 0.56	0.5 Meters per second Standard Deviation 0.41	-0.4 Meters per second Standard Deviation 0.39	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=85 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=22 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=63 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in 10 Meter Walk or Run Velocity at Month 30: Ambulatory Participants Aged >=3 Years	-0.3 Meters per second Standard Deviation 0.62	0.4 Meters per second Standard Deviation 0.52	-0.6 Meters per second Standard Deviation 0.44	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=181 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=23 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=158 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=5 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=186 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years Left Knee Extension	0.1 Kilograms Standard Deviation 1.50	0.5 Kilograms Standard Deviation 1.91	0.0 Kilograms Standard Deviation 1.43	0.3 Kilograms Standard Deviation 0.29	0.1 Kilograms Standard Deviation 1.49
Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years Right Knee Extension	0.0 Kilograms Standard Deviation 1.87	0.7 Kilograms Standard Deviation 2.35	-0.1 Kilograms Standard Deviation 1.78	-0.0 Kilograms Standard Deviation 0.37	0.0 Kilograms Standard Deviation 1.85

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=175 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=18 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=157 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=28 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=203 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years Left Knee Extension	-0.1 Kilograms Standard Deviation 1.80	-0.1 Kilograms Standard Deviation 1.25	-0.1 Kilograms Standard Deviation 1.85	-0.3 Kilograms Standard Deviation 1.31	-0.1 Kilograms Standard Deviation 1.74
Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years Right Knee Extension	-0.4 Kilograms Standard Deviation 1.84	0.1 Kilograms Standard Deviation 1.50	-0.4 Kilograms Standard Deviation 1.87	-0.3 Kilograms Standard Deviation 1.10	-0.4 Kilograms Standard Deviation 1.75

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=129 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=18 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=111 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=133 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years Left Knee Extension	-0.3 Kilograms Standard Deviation 1.95	0.7 Kilograms Standard Deviation 1.69	-0.4 Kilograms Standard Deviation 1.95	-0.7 Kilograms Standard Deviation 1.05	-0.3 Kilograms Standard Deviation 1.93
Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years Right Knee Extension	-0.6 Kilograms Standard Deviation 2.28	0.5 Kilograms Standard Deviation 1.96	-0.7 Kilograms Standard Deviation 2.29	-0.8 Kilograms Standard Deviation 0.42	-0.6 Kilograms Standard Deviation 2.25

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=155 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=136 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=178 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years Left Knee Extension	-0.6 Kilograms Standard Deviation 2.00	-0.4 Kilograms Standard Deviation 2.37	-0.6 Kilograms Standard Deviation 1.95	-0.5 Kilograms Standard Deviation 1.64	-0.6 Kilograms Standard Deviation 1.95
Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years Right Knee Extension	-0.9 Kilograms Standard Deviation 2.23	0.0 Kilograms Standard Deviation 2.77	-1.0 Kilograms Standard Deviation 2.13	-0.7 Kilograms Standard Deviation 1.62	-0.8 Kilograms Standard Deviation 2.16

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right knee extension was analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=83 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=7 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=76 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=3 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=86 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years Left Knee Extension	-0.7 Kilograms Standard Deviation 2.60	-0.9 Kilograms Standard Deviation 2.12	-0.7 Kilograms Standard Deviation 2.66	-0.4 Kilograms Standard Deviation 0.36	-0.7 Kilograms Standard Deviation 2.56
Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years Right Knee Extension	-1.4 Kilograms Standard Deviation 2.76	-2.3 Kilograms Standard Deviation 2.89	-1.4 Kilograms Standard Deviation 2.76	-1.1 Kilograms Standard Deviation 0.44	-1.4 Kilograms Standard Deviation 2.72

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Muscle strength was recorded by handheld myometry. Left and right elbow flexion were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=184 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=24 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=160 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=5 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=189 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years Left Elbow Flexion	0.3 Kilograms Standard Deviation 0.92	0.3 Kilograms Standard Deviation 0.85	0.3 Kilograms Standard Deviation 0.93	-0.4 Kilograms Standard Deviation 0.30	0.3 Kilograms Standard Deviation 0.92
Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years Right Elbow Flexion	0.3 Kilograms Standard Deviation 0.91	0.4 Kilograms Standard Deviation 0.81	0.3 Kilograms Standard Deviation 0.93	-0.3 Kilograms Standard Deviation 0.25	0.3 Kilograms Standard Deviation 0.91

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Outcome measures

Outcome measures
Measure	Total Participants n=178 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=159 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=207 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years Left Elbow Flexion	0.2 Kilograms Standard Deviation 1.02	0.5 Kilograms Standard Deviation 0.97	0.1 Kilograms Standard Deviation 1.02	0.1 Kilograms Standard Deviation 1.12	0.1 Kilograms Standard Deviation 1.03
Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years Right Elbow Flexion	0.3 Kilograms Standard Deviation 1.05	0.5 Kilograms Standard Deviation 0.79	0.2 Kilograms Standard Deviation 1.07	-0.1 Kilograms Standard Deviation 0.89	0.2 Kilograms Standard Deviation 1.03

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Outcome measures

Outcome measures
Measure	Total Participants n=133 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=114 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=137 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years Left Elbow Flexion	0.2 Kilograms Standard Deviation 1.11	0.7 Kilograms Standard Deviation 0.80	0.2 Kilograms Standard Deviation 1.14	-1.0 Kilograms Standard Deviation 0.83	0.2 Kilograms Standard Deviation 1.12
Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years Right Elbow Flexion	0.3 Kilograms Standard Deviation 1.12	0.5 Kilograms Standard Deviation 0.85	0.2 Kilograms Standard Deviation 1.16	-0.9 Kilograms Standard Deviation 1.35	0.2 Kilograms Standard Deviation 1.13

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=157 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=20 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=137 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=180 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years Left Elbow Flexion	0.1 Kilograms Standard Deviation 1.17	0.8 Kilograms Standard Deviation 0.89	0.0 Kilograms Standard Deviation 1.18	-0.0 Kilograms Standard Deviation 0.92	0.1 Kilograms Standard Deviation 1.14
Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years Right Elbow Flexion	0.2 Kilograms Standard Deviation 1.15	0.9 Kilograms Standard Deviation 0.81	0.1 Kilograms Standard Deviation 1.16	-0.3 Kilograms Standard Deviation 1.15	0.1 Kilograms Standard Deviation 1.16

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=84 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=7 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=77 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=88 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years Right Elbow Flexion	0.4 Kilograms Standard Deviation 1.50	0.4 Kilograms Standard Deviation 0.70	0.4 Kilograms Standard Deviation 1.56	-0.0 Kilograms Standard Deviation 0.68	0.4 Kilograms Standard Deviation 1.47
Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years Left Elbow Flexion	0.2 Kilograms Standard Deviation 1.53	0.8 Kilograms Standard Deviation 0.72	0.2 Kilograms Standard Deviation 1.58	-0.0 Kilograms Standard Deviation 0.83	0.2 Kilograms Standard Deviation 1.51

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Muscle strength was recorded by handheld myometry. Left and right elbow extension were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=183 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=23 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=160 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=5 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years Left Elbow Extension	0.2 Kilograms Standard Deviation 0.79	0.4 Kilograms Standard Deviation 0.85	0.2 Kilograms Standard Deviation 0.78	0.1 Kilograms Standard Deviation 0.21	0.2 Kilograms Standard Deviation 0.78
Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years Right Elbow Extension	0.3 Kilograms Standard Deviation 0.92	0.3 Kilograms Standard Deviation 1.22	0.3 Kilograms Standard Deviation 0.87	0.1 Kilograms Standard Deviation 0.26	0.3 Kilograms Standard Deviation 0.91

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=177 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=18 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=159 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=206 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years Left Elbow Extension	0.2 Kilograms Standard Deviation 0.85	0.2 Kilograms Standard Deviation 0.74	0.2 Kilograms Standard Deviation 0.87	0.0 Kilograms Standard Deviation 0.68	0.2 Kilograms Standard Deviation 0.83
Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years Right Elbow Extension	0.3 Kilograms Standard Deviation 0.96	0.4 Kilograms Standard Deviation 0.88	0.3 Kilograms Standard Deviation 0.97	0.2 Kilograms Standard Deviation 0.76	0.3 Kilograms Standard Deviation 0.93

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=132 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=18 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=114 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=136 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years Left Elbow Extension	0.2 Kilograms Standard Deviation 0.99	0.6 Kilograms Standard Deviation 0.80	0.2 Kilograms Standard Deviation 1.00	-0.4 Kilograms Standard Deviation 0.73	0.2 Kilograms Standard Deviation 0.98
Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years Right Elbow Extension	0.2 Kilograms Standard Deviation 1.06	-0.0 Kilograms Standard Deviation 0.84	0.2 Kilograms Standard Deviation 1.09	-0.2 Kilograms Standard Deviation 0.13	0.2 Kilograms Standard Deviation 1.04

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=156 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=137 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=179 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years Left Elbow Extension	0.3 Kilograms Standard Deviation 1.08	1.1 Kilograms Standard Deviation 1.14	0.1 Kilograms Standard Deviation 1.03	-0.1 Kilograms Standard Deviation 0.73	0.2 Kilograms Standard Deviation 1.05
Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years Right Elbow Extension	0.1 Kilograms Standard Deviation 1.13	0.7 Kilograms Standard Deviation 1.29	0.0 Kilograms Standard Deviation 1.09	0.3 Kilograms Standard Deviation 0.96	0.1 Kilograms Standard Deviation 1.11

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=84 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=7 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=77 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=88 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years Left Elbow Extension	0.3 Kilograms Standard Deviation 1.32	0.8 Kilograms Standard Deviation 1.55	0.2 Kilograms Standard Deviation 1.30	-0.3 Kilograms Standard Deviation 0.79	0.3 Kilograms Standard Deviation 1.30
Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years Right Elbow Extension	0.2 Kilograms Standard Deviation 1.36	-0.9 Kilograms Standard Deviation 0.72	0.3 Kilograms Standard Deviation 1.37	-0.0 Kilograms Standard Deviation 0.37	0.2 Kilograms Standard Deviation 1.33

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Muscle strength was recorded by handheld myometry. Left and right shoulder abduction were analyzed. The muscle strength test was only performed in participants \>=5 years old as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=183 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=24 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=159 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=5 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years Left Shoulder Abduction	0.4 Kilograms Standard Deviation 0.91	0.4 Kilograms Standard Deviation 1.15	0.4 Kilograms Standard Deviation 0.87	-0.1 Kilograms Standard Deviation 0.45	0.4 Kilograms Standard Deviation 0.90
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years Right Shoulder Abduction	0.4 Kilograms Standard Deviation 0.90	0.5 Kilograms Standard Deviation 0.95	0.4 Kilograms Standard Deviation 0.90	0.3 Kilograms Standard Deviation 0.10	0.4 Kilograms Standard Deviation 0.89

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Outcome measures

Outcome measures
Measure	Total Participants n=177 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=158 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=206 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years Left Shoulder Abduction	0.4 Kilograms Standard Deviation 0.88	0.5 Kilograms Standard Deviation 0.76	0.4 Kilograms Standard Deviation 0.89	0.1 Kilograms Standard Deviation 0.82	0.4 Kilograms Standard Deviation 0.88
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years Right Shoulder Abduction	0.5 Kilograms Standard Deviation 0.99	0.6 Kilograms Standard Deviation 0.86	0.5 Kilograms Standard Deviation 1.00	0.1 Kilograms Standard Deviation 0.97	0.4 Kilograms Standard Deviation 0.99

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Outcome measures

Outcome measures
Measure	Total Participants n=132 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=19 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=113 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=136 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years Right Shoulder Abduction	0.5 Kilograms Standard Deviation 1.04	0.8 Kilograms Standard Deviation 0.98	0.4 Kilograms Standard Deviation 1.05	-0.8 Kilograms Standard Deviation 0.55	0.4 Kilograms Standard Deviation 1.05
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years Left Shoulder Abduction	0.4 Kilograms Standard Deviation 0.94	0.5 Kilograms Standard Deviation 0.65	0.4 Kilograms Standard Deviation 0.98	-1.0 Kilograms Standard Deviation 0.60	0.3 Kilograms Standard Deviation 0.96

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Outcome measures

Outcome measures
Measure	Total Participants n=156 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=20 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=136 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=179 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years Left Shoulder Abduction	0.4 Kilograms Standard Deviation 1.15	1.1 Kilograms Standard Deviation 1.04	0.3 Kilograms Standard Deviation 1.13	0.0 Kilograms Standard Deviation 0.80	0.4 Kilograms Standard Deviation 1.12
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years Right Shoulder Abduction	0.6 Kilograms Standard Deviation 1.24	1.2 Kilograms Standard Deviation 1.11	0.6 Kilograms Standard Deviation 1.25	-0.1 Kilograms Standard Deviation 1.04	0.5 Kilograms Standard Deviation 1.24

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=83 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=7 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=76 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=87 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years Left Shoulder Abduction	0.6 Kilograms Standard Deviation 1.25	0.6 Kilograms Standard Deviation 1.28	0.6 Kilograms Standard Deviation 1.25	0.8 Kilograms Standard Deviation 1.00	0.6 Kilograms Standard Deviation 1.23
Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years Right Shoulder Abduction	0.8 Kilograms Standard Deviation 1.31	0.8 Kilograms Standard Deviation 0.93	0.8 Kilograms Standard Deviation 1.34	1.3 Kilograms Standard Deviation 1.77	0.8 Kilograms Standard Deviation 1.32

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Outcome measures

Outcome measures
Measure	Total Participants n=256 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=94 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=162 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=5 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=261 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6 Right Ankle Joint	-0.7 Degrees of passive dorsiflexion Standard Deviation 9.96	-1.2 Degrees of passive dorsiflexion Standard Deviation 8.59	-0.4 Degrees of passive dorsiflexion Standard Deviation 10.69	-8.2 Degrees of passive dorsiflexion Standard Deviation 5.45	-0.8 Degrees of passive dorsiflexion Standard Deviation 9.94
Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6 Left Ankle Joint	0.3 Degrees of passive dorsiflexion Standard Deviation 10.31	-0.5 Degrees of passive dorsiflexion Standard Deviation 9.12	0.8 Degrees of passive dorsiflexion Standard Deviation 10.94	-8.2 Degrees of passive dorsiflexion Standard Deviation 8.50	0.2 Degrees of passive dorsiflexion Standard Deviation 10.33

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Outcome measures

Outcome measures
Measure	Total Participants n=240 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=80 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=160 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=28 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=268 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Ankles at Month 12 Left Ankle Joint	-2.2 Degrees of passive dorsiflexion Standard Deviation 10.74	-2.6 Degrees of passive dorsiflexion Standard Deviation 10.13	-2.0 Degrees of passive dorsiflexion Standard Deviation 11.06	-11.6 Degrees of passive dorsiflexion Standard Deviation 18.18	-3.2 Degrees of passive dorsiflexion Standard Deviation 12.05
Change From Baseline in ROM at Bilateral Ankles at Month 12 Right Ankle Joint	-2.9 Degrees of passive dorsiflexion Standard Deviation 10.31	-3.1 Degrees of passive dorsiflexion Standard Deviation 9.19	-2.8 Degrees of passive dorsiflexion Standard Deviation 10.85	-7.9 Degrees of passive dorsiflexion Standard Deviation 19.74	-3.4 Degrees of passive dorsiflexion Standard Deviation 11.70

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Outcome measures

Outcome measures
Measure	Total Participants n=192 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=77 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=115 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=196 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Ankles at Month 18 Left Ankle Joint	-3.1 Degrees of passive dorsiflexion Standard Deviation 10.28	-1.8 Degrees of passive dorsiflexion Standard Deviation 9.19	-4.0 Degrees of passive dorsiflexion Standard Deviation 10.90	-8.8 Degrees of passive dorsiflexion Standard Deviation 12.47	-3.2 Degrees of passive dorsiflexion Standard Deviation 10.32
Change From Baseline in ROM at Bilateral Ankles at Month 18 Right Ankle Joint	-3.6 Degrees of passive dorsiflexion Standard Deviation 10.58	-2.2 Degrees of passive dorsiflexion Standard Deviation 8.91	-4.5 Degrees of passive dorsiflexion Standard Deviation 11.50	-5.0 Degrees of passive dorsiflexion Standard Deviation 4.24	-3.6 Degrees of passive dorsiflexion Standard Deviation 10.48

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Outcome measures

Outcome measures
Measure	Total Participants n=211 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=136 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=234 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Ankles at Month 24 Left Ankle Joint	-4.1 Degrees of passive dorsiflexion Standard Deviation 11.00	-2.8 Degrees of passive dorsiflexion Standard Deviation 8.59	-4.8 Degrees of passive dorsiflexion Standard Deviation 12.09	-8.5 Degrees of passive dorsiflexion Standard Deviation 15.79	-4.6 Degrees of passive dorsiflexion Standard Deviation 11.59
Change From Baseline in ROM at Bilateral Ankles at Month 24 Right Ankle Joint	-5.1 Degrees of passive dorsiflexion Standard Deviation 11.62	-3.6 Degrees of passive dorsiflexion Standard Deviation 8.78	-6.0 Degrees of passive dorsiflexion Standard Deviation 12.88	-11.4 Degrees of passive dorsiflexion Standard Deviation 15.77	-5.8 Degrees of passive dorsiflexion Standard Deviation 12.20

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of ankle contractures. The ROM at left and right ankles were measured in degrees of passive dorsiflexion.

Outcome measures

Outcome measures
Measure	Total Participants n=106 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=30 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=76 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=110 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Ankles at Month 30 Left Ankle Joint	-3.4 Degrees of passive dorsiflexion Standard Deviation 11.71	0.3 Degrees of passive dorsiflexion Standard Deviation 9.50	-4.9 Degrees of passive dorsiflexion Standard Deviation 12.23	-4.8 Degrees of passive dorsiflexion Standard Deviation 14.08	-3.5 Degrees of passive dorsiflexion Standard Deviation 11.73
Change From Baseline in ROM at Bilateral Ankles at Month 30 Right Ankle Joint	-4.6 Degrees of passive dorsiflexion Standard Deviation 12.18	0.1 Degrees of passive dorsiflexion Standard Deviation 8.08	-6.5 Degrees of passive dorsiflexion Standard Deviation 13.04	-2.5 Degrees of passive dorsiflexion Standard Deviation 6.24	-4.5 Degrees of passive dorsiflexion Standard Deviation 12.01

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 6

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Outcome measures

Outcome measures
Measure	Total Participants n=256 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=94 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=162 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=5 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=261 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Elbows at Month 6 Right Elbow Joint	1.0 Degrees of passive extension Standard Deviation 6.92	1.0 Degrees of passive extension Standard Deviation 7.13	1.0 Degrees of passive extension Standard Deviation 6.82	-3.2 Degrees of passive extension Standard Deviation 7.79	0.9 Degrees of passive extension Standard Deviation 6.95
Change From Baseline in ROM at Bilateral Elbows at Month 6 Left Elbow Joint	0.8 Degrees of passive extension Standard Deviation 6.53	0.8 Degrees of passive extension Standard Deviation 7.13	0.7 Degrees of passive extension Standard Deviation 6.17	-4.0 Degrees of passive extension Standard Deviation 2.92	0.7 Degrees of passive extension Standard Deviation 6.51

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Outcome measures

Outcome measures
Measure	Total Participants n=240 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=80 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=160 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=269 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Elbows at Month 12 Right Elbow Joint	-1.1 Degrees of passive extension Standard Deviation 8.63	-2.7 Degrees of passive extension Standard Deviation 9.86	-0.3 Degrees of passive extension Standard Deviation 7.85	-2.7 Degrees of passive extension Standard Deviation 11.19	-1.3 Degrees of passive extension Standard Deviation 8.93
Change From Baseline in ROM at Bilateral Elbows at Month 12 Left Elbow Joint	-1.6 Degrees of passive extension Standard Deviation 8.77	-2.3 Degrees of passive extension Standard Deviation 10.33	-1.3 Degrees of passive extension Standard Deviation 7.90	-3.3 Degrees of passive extension Standard Deviation 11.52	-1.8 Degrees of passive extension Standard Deviation 9.10

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 18

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Outcome measures

Outcome measures
Measure	Total Participants n=192 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=77 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=115 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=196 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Elbows at Month 18 Right Elbow Joint	-1.7 Degrees of passive extension Standard Deviation 8.18	-1.6 Degrees of passive extension Standard Deviation 8.44	-1.8 Degrees of passive extension Standard Deviation 8.03	-7.0 Degrees of passive extension Standard Deviation 12.99	-1.8 Degrees of passive extension Standard Deviation 8.28
Change From Baseline in ROM at Bilateral Elbows at Month 18 Left Elbow Joint	-2.0 Degrees of passive extension Standard Deviation 7.30	-1.1 Degrees of passive extension Standard Deviation 8.43	-2.6 Degrees of passive extension Standard Deviation 6.41	-10.3 Degrees of passive extension Standard Deviation 2.63	-2.1 Degrees of passive extension Standard Deviation 7.33

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Outcome measures

Outcome measures
Measure	Total Participants n=212 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=137 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=235 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Elbows at Month 24 Right Elbow Joint	-0.6 Degrees of passive extension Standard Deviation 7.17	0.1 Degrees of passive extension Standard Deviation 6.68	-1.0 Degrees of passive extension Standard Deviation 7.42	-5.1 Degrees of passive extension Standard Deviation 13.22	-1.1 Degrees of passive extension Standard Deviation 8.04
Change From Baseline in ROM at Bilateral Elbows at Month 24 Left Elbow Joint	-0.7 Degrees of passive extension Standard Deviation 6.98	0.7 Degrees of passive extension Standard Deviation 6.79	-1.5 Degrees of passive extension Standard Deviation 6.99	-5.7 Degrees of passive extension Standard Deviation 13.87	-1.2 Degrees of passive extension Standard Deviation 8.02

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Range of motion was evaluated by using goniometry to record any occurrences of elbow contractures. The ROM at left and right elbows were measured in degrees of passive extension.

Outcome measures

Outcome measures
Measure	Total Participants n=107 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=30 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=77 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=111 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in ROM at Bilateral Elbows at Month 30 Right Elbow Joint	-1.1 Degrees of passive extension Standard Deviation 7.10	1.0 Degrees of passive extension Standard Deviation 5.47	-1.9 Degrees of passive extension Standard Deviation 7.52	-5.0 Degrees of passive extension Standard Deviation 17.74	-1.2 Degrees of passive extension Standard Deviation 7.60
Change From Baseline in ROM at Bilateral Elbows at Month 30 Left Elbow Joint	-1.3 Degrees of passive extension Standard Deviation 7.19	1.0 Degrees of passive extension Standard Deviation 6.69	-2.2 Degrees of passive extension Standard Deviation 7.23	-9.0 Degrees of passive extension Standard Deviation 14.26	-1.5 Degrees of passive extension Standard Deviation 7.58

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Forced vital capacity (FVC) is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=158 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=30 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Predicted Forced Vital Capacity (%pFVC) at Month 12: Participants Aged >=6 Years	-3.4 Percent predicted FVC Standard Deviation 16.52	-2.9 Percent predicted FVC Standard Deviation 15.85	-5.7 Percent predicted FVC Standard Deviation 19.79	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

FVC is the volume of air that can be maximally forcefully exhaled after taking the deepest breath possible and was measured using spirometry. The percent predicted FVC was calculated from FVC (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for spirometry. The pulmonary function assessments were performed in participants aged \>=6 years as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=158 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=135 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years	-5.7 Percent predicted FVC Standard Deviation 17.02	-4.3 Percent predicted FVC Standard Deviation 15.97	-14.1 Percent predicted FVC Standard Deviation 20.68	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=79 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years	99.9 Percent predicted FVC Standard Deviation 950.53	105.4 Percent predicted FVC Standard Deviation 975.57	-3.2 Percent predicted FVC Standard Deviation 9.41	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=158 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=30 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFVC at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	-3.4 Percent predicted FVC Standard Deviation 16.52	-2.9 Percent predicted FVC Standard Deviation 15.85	-5.7 Percent predicted FVC Standard Deviation 19.79	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=158 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=135 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	-5.7 Percent predicted FVC Standard Deviation 17.02	-4.3 Percent predicted FVC Standard Deviation 15.97	-14.1 Percent predicted FVC Standard Deviation 20.68	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=78 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=74 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	-7.0 Percent predicted FVC Standard Deviation 16.12	-7.2 Percent predicted FVC Standard Deviation 16.41	-3.2 Percent predicted FVC Standard Deviation 9.41	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Forced expiratory volume in one second (FEV1) is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=158 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=30 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%pFEV1) at Month 12: Participants Aged >=6 Years	-2.1 Percent predicted FEV1 Standard Deviation 18.78	-1.8 Percent predicted FEV1 Standard Deviation 18.65	-4.0 Percent predicted FEV1 Standard Deviation 19.64	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

FEV1 is the volume of air forcefully exhaled in 1 second and was measured using spirometry. The %pFEV1 was calculated from FEV1 (measured in liter) according to age, height (estimated height as derived from the ulna length for non-ambulatory participants), ethnicity, and gender using multi-ethnic reference values for Spirometry. The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=158 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=135 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years	-4.8 Percent predicted FEV1 Standard Deviation 19.67	-3.9 Percent predicted FEV1 Standard Deviation 19.38	-10.2 Percent predicted FEV1 Standard Deviation 20.94	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=79 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years	109.1 Percent predicted FEV1 Standard Deviation 1010.75	115.2 Percent predicted FEV1 Standard Deviation 1037.35	-5.8 Percent predicted FEV1 Standard Deviation 8.83	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=158 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=30 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFEV1 at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	-2.1 Percent predicted FEV1 Standard Deviation 18.78	-1.8 Percent predicted FEV1 Standard Deviation 18.65	-4.0 Percent predicted FEV1 Standard Deviation 19.64	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=158 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=135 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	-4.8 Percent predicted FEV1 Standard Deviation 19.67	-3.9 Percent predicted FEV1 Standard Deviation 19.38	-10.2 Percent predicted FEV1 Standard Deviation 20.94	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=78 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=74 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	-4.6 Percent predicted FEV1 Standard Deviation 18.56	-4.6 Percent predicted FEV1 Standard Deviation 18.97	-5.8 Percent predicted FEV1 Standard Deviation 8.83	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=169 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=141 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=28 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Maximum Inspiratory Pressure at Month 12: Participants Aged >=6 Years	0.6 Centimeter of water (cm H2O) Standard Deviation 18.62	1.4 Centimeter of water (cm H2O) Standard Deviation 18.22	-3.1 Centimeter of water (cm H2O) Standard Deviation 20.46	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=145 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=122 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Maximum Inspiratory Pressure at Month 24: Participants Aged >=6 Years	0.3 Centimeter of water (cm H2O) Standard Deviation 18.08	0.9 Centimeter of water (cm H2O) Standard Deviation 18.24	-2.8 Centimeter of water (cm H2O) Standard Deviation 17.24	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=71 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=67 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Maximum Inspiratory Pressure at Month 30: Participants Aged >=6 Years	1.6 Centimeter of water (cm H2O) Standard Deviation 19.22	2.5 Centimeter of water (cm H2O) Standard Deviation 19.19	-14.0 Centimeter of water (cm H2O) Standard Deviation 13.22	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

Outcome measures

Outcome measures
Measure	Total Participants n=183 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=154 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Maximum Expiratory Pressure at Month 12: Participants Aged >=6 Years	-0.0 Centimeter of water (cm H2O) Standard Deviation 14.80	1.0 Centimeter of water (cm H2O) Standard Deviation 14.72	-5.5 Centimeter of water (cm H2O) Standard Deviation 14.24	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

Outcome measures

Outcome measures
Measure	Total Participants n=155 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=132 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Maximum Expiratory Pressure at Month 24: Participants Aged >=6 Years	0.1 Centimeter of water (cm H2O) Standard Deviation 17.48	0.6 Centimeter of water (cm H2O) Standard Deviation 18.07	-2.5 Centimeter of water (cm H2O) Standard Deviation 13.67	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in SAP.

Outcome measures

Outcome measures
Measure	Total Participants n=79 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=75 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Maximum Expiratory Pressure at Month 30: Participants Aged >=6 Years	0.6 Centimeter of water (cm H2O) Standard Deviation 18.68	1.7 Centimeter of water (cm H2O) Standard Deviation 17.91	-20.3 Centimeter of water (cm H2O) Standard Deviation 23.61	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=187 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=157 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=30 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years	17.9 Liters per minute Standard Deviation 49.94	14.0 Liters per minute Standard Deviation 46.61	38.6 Liters per minute Standard Deviation 61.50	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=156 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=133 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years	24.4 Liters per minute Standard Deviation 60.84	20.7 Liters per minute Standard Deviation 59.04	46.1 Liters per minute Standard Deviation 67.78	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=78 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=74 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years	27.7 Liters per minute Standard Deviation 62.32	25.2 Liters per minute Standard Deviation 62.83	74.0 Liters per minute Standard Deviation 24.84	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Outcome measures

Outcome measures
Measure	Total Participants n=187 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=157 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=30 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	17.9 Liters per minute Standard Deviation 49.94	14.0 Liters per minute Standard Deviation 46.61	38.6 Liters per minute Standard Deviation 61.50	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Outcome measures

Outcome measures
Measure	Total Participants n=155 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=132 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=23 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	25.9 Liters per minute Standard Deviation 58.22	22.4 Liters per minute Standard Deviation 55.94	46.1 Liters per minute Standard Deviation 67.78	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

The pulmonary function assessments were performed only in participants \>=6 years old as pre-specified in protocol. Incorrect data were addressed by unplanned analysis.

Outcome measures

Outcome measures
Measure	Total Participants n=78 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=74 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	27.7 Liters per minute Standard Deviation 62.32	25.2 Liters per minute Standard Deviation 62.83	74.0 Liters per minute Standard Deviation 24.84	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

LVEF was the percentage of blood that was ejected out of left ventricle with each contraction, estimated by echocardiography. The LVEF was only performed in participants \>=6 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=189 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=160 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 12: Participants Aged >=6 Years	3.4 Percentage of ejected blood Standard Deviation 41.83	4.1 Percentage of ejected blood Standard Deviation 45.37	-0.4 Percentage of ejected blood Standard Deviation 6.32	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=161 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=137 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=24 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years	-0.8 Percentage of ejected blood Standard Deviation 5.78	-0.7 Percentage of ejected blood Standard Deviation 5.43	-1.6 Percentage of ejected blood Standard Deviation 7.58	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=82 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=78 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years	-0.4 Percentage of ejected blood Standard Deviation 6.34	-0.3 Percentage of ejected blood Standard Deviation 6.42	-2.0 Percentage of ejected blood Standard Deviation 4.69	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=188 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=159 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=29 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in LVEF at Month 12: Participants Aged >=6 Years (Unplanned Analysis)	0.4 Percentage of ejected blood Standard Deviation 6.27	0.5 Percentage of ejected blood Standard Deviation 6.27	-0.4 Percentage of ejected blood Standard Deviation 6.32	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=161 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=137 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=24 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years (Unplanned Analysis)	-0.8 Percentage of ejected blood Standard Deviation 5.78	-0.7 Percentage of ejected blood Standard Deviation 5.43	-1.6 Percentage of ejected blood Standard Deviation 7.58	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=82 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=78 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years (Unplanned Analysis)	-0.4 Percentage of ejected blood Standard Deviation 6.34	-0.3 Percentage of ejected blood Standard Deviation 6.42	-2.0 Percentage of ejected blood Standard Deviation 4.69	—	—

PRIMARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

WISC-IV is an individually administered intelligence test for children between the ages of 6 and 16. The WISC-IV Composites are: Verbal Comprehension, Perceptual Reasoning, Working Memory, and Processing Speed. Scores from the Composites constitute the WISC-IV Full Scale IQ score which ranges from 40 (Exceptionally Low) to 160 (Exceptionally Superior), higher scores indicated more intelligence. The WISC was only performed in ambulatory participants \>= 6 to \<=16 years old as pre-specified in the protocol.

Outcome measures

Outcome measures
Measure	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=97 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Wechsler Intelligence Scale for Children (WISC)-IV Score at Month 24: Ambulatory Participants >= 6 to <=16 Years	—	1.0 Units on a scale Standard Deviation 8.59	—	—	—

SECONDARY outcome

Timeframe: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study.

Number of participants as per type of mutation: exon deletion, exon duplication, point mutation, small insertion, small deletion and others is presented in this outcome measure. One participant could have more than 1 mutation type.

Outcome measures

Outcome measures
Measure	Total Participants n=36 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=312 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants With Type of DMD Mutation Exon Deletion	18 Participants	64 Participants	127 Participants	209 Participants	—
Number of Participants With Type of DMD Mutation Exon Duplication	6 Participants	10 Participants	21 Participants	37 Participants	—
Number of Participants With Type of DMD Mutation Point Mutation	8 Participants	18 Participants	21 Participants	47 Participants	—
Number of Participants With Type of DMD Mutation Small Insertion	3 Participants	1 Participants	1 Participants	5 Participants	—
Number of Participants With Type of DMD Mutation Small Deletion	1 Participants	5 Participants	7 Participants	13 Participants	—
Number of Participants With Type of DMD Mutation Others	0 Participants	2 Participants	1 Participants	3 Participants	—

SECONDARY outcome

Timeframe: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set with affected exon. The frequency (count) of each affected exon by mutation types was defined to be plotted for 3 groups combined, because the mutation types/affected exons were not anticipated to be varied in different age groups or by ambulation status according to available evidence globally.

Number of participants with each affected exon by mutation types is presented in this outcome measure. Only those categories with non-zero values have been reported.

Outcome measures

Outcome measures
Measure	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=299 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants With Each Affected Exon by Mutation Types Exon 50, Exon deletion	—	94 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 44, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 45, Exon deletion	—	42 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 45, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 46, Exon deletion	—	73 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 46, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 46, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 47, Exon deletion	—	73 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 47, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 47, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 47, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 48, Exon deletion	—	83 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 48, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 48, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 1, Exon deletion	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 2, Exon deletion	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 2, Exon duplication	—	6 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 3, Exon deletion	—	16 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 3, Exon duplication	—	5 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 6, Exon deletion	—	20 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 6, Exon duplication	—	8 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 6, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 6, Others	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 7, Exon deletion	—	20 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 7, Exon duplication	—	8 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 8, Exon deletion	—	21 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 8, Exon duplication	—	6 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 9, Exon deletion	—	21 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 9, Exon duplication	—	5 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 10, Exon deletion	—	17 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 10, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 10, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 11, Exon deletion	—	17 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 11, Exon duplication	—	5 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 12, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 13, Exon deletion	—	17 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 13, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 13, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 14, Exon deletion	—	17 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 14, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 15, Exon deletion	—	17 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 15, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 15, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 16, Exon deletion	—	18 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 16, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 17, Exon deletion	—	20 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 17, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 18, Exon deletion	—	15 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 18, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 20, Exon deletion	—	13 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 20, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 20, Point Mutation	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 21, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 21, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 21, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 22, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 22, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 22, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 23, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 23, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 23, Point Mutation	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 25, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 25, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 26, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 26, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 26, Point Mutation	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 26, Small insertion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 26, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 27, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 27, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 27, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 27, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 28, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 28, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 29, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 29, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 29, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 32, Point Mutation	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 32, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 33, Exon deletion	—	9 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 33, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 33, Point Mutation	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 34, Exon deletion	—	8 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 34, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 34, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 35, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 35, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 37, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 37, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 37, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 38, Exon deletion	—	8 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 38, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 49, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 38, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 39, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 39, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 39, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 39, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 39, Others	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 40, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 40, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 40, Small insertion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 41, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 41, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 41, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 42, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 42, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 42, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 43, Exon deletion	—	9 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 43, Exon duplication	—	5 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 43, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 44, Exon deletion	—	20 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 44, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 44, Point Mutation	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 53, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 53, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 54, Exon deletion	—	22 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 54, Exon duplication	—	4 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 55, Exon deletion	—	6 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 56, Small deletion	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 57, Exon deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 57, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 57, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 58, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 58, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 58, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 59, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 59, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 60, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 61, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 62, Exon duplication	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 62, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 63, Exon deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 63, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 70, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 70, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 71, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 72, Exon duplication	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 73, Exon duplication	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 74, Exon duplication	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 4, Exon deletion	—	16 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 4, Exon duplication	—	5 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 5, Exon deletion	—	21 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 5, Exon duplication	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 5, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 12, Exon deletion	—	16 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 19, Exon deletion	—	14 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 19, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 19, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 24, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 24, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 24, Point Mutation	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 24, Small insertion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 25, Exon deletion	—	12 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 25, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 28, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 30, Exon deletion	—	9 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 30, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 30, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 31, Exon deletion	—	9 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 31, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 31, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 32, Exon deletion	—	9 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 32, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 33, Small deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 35, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 36, Exon deletion	—	7 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 36, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 36, Point Mutation	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 40, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 48, Small insertion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 49, Exon deletion	—	96 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 49, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 50, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 51, Exon deletion	—	64 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 51, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 52, Exon deletion	—	56 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 52, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 53, Exon deletion	—	24 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 55, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 55, Point Mutation	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 56, Exon deletion	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 56, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 64, Exon duplication	—	2 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 65, Exon deletion	—	1 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 65, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 66, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 67, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 68, Exon duplication	—	3 Participants	—	—	—
Number of Participants With Each Affected Exon by Mutation Types Exon 69, Exon duplication	—	2 Participants	—	—	—

SECONDARY outcome

Timeframe: Up to Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study.

Number of participants with DMD mutations affecting any exon between exon 9 and exon 13 or deletion that affects both exon 29 and exon 30 is presented in this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=36 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=312 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants With DMD Mutations Affecting Any Exon Between Exon 9 and Exon 13 or Deletion That Affects Both Exon 29 and Exon 30 Deletion affects both exon 29 and exon 30	1 Participants	2 Participants	6 Participants	9 Participants	—
Number of Participants With DMD Mutations Affecting Any Exon Between Exon 9 and Exon 13 or Deletion That Affects Both Exon 29 and Exon 30 Any mutation affecting any exon between exon 9 and exon 13	2 Participants	11 Participants	20 Participants	33 Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies all participants in the Full Analysis Set with age \<=10 years contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

The PODCI is a participant-reported assessment of musculoskeletal health intended for use in children and adolescents. The pediatric version was intended for completion by parents or caregivers of children \<=10 years old. It included a Global Function Scale along with core scales: Upper Extremity and Physical Function Core Scale (8 items); Transfer and Basic Mobility Core Scale (11 items); Sports and Physical Functioning Core Scale (21 items); Pain/Comfort Core Scale (3 items); Happiness Core Scale (5 items). The scores of each PODCI global functioning scale and core scales are averaged over the number of items answered (omitted no entry items). Mean of the rescaled values is multiplied by a constant so that each global functioning scale and core scales has a final range of values between 0-100. The higher scores represent less disability and better functioning.

Outcome measures

Outcome measures
Measure	Total Participants n=245 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=146 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=9 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=254 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 6	0.7 Units on a scale Standard Deviation 8.47	1.2 Units on a scale Standard Deviation 9.58	0.3 Units on a scale Standard Deviation 7.61	16.5 Units on a scale Standard Deviation 12.02	0.8 Units on a scale Standard Deviation 8.61
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 12	-0.6 Units on a scale Standard Deviation 11.01	0.7 Units on a scale Standard Deviation 10.90	-1.4 Units on a scale Standard Deviation 11.05	-8.3 Units on a scale Standard Deviation 17.56	-0.7 Units on a scale Standard Deviation 11.17
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 18	0.8 Units on a scale Standard Deviation 9.85	2.0 Units on a scale Standard Deviation 10.54	-0.3 Units on a scale Standard Deviation 9.13	—	0.8 Units on a scale Standard Deviation 9.85
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 24	-2.5 Units on a scale Standard Deviation 11.71	-0.8 Units on a scale Standard Deviation 11.41	-4.1 Units on a scale Standard Deviation 11.83	—	-2.5 Units on a scale Standard Deviation 11.71
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 18	-2.8 Units on a scale Standard Deviation 15.83	2.6 Units on a scale Standard Deviation 14.05	-7.5 Units on a scale Standard Deviation 15.88	—	-2.8 Units on a scale Standard Deviation 15.83
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 30	0.6 Units on a scale Standard Deviation 10.85	4.1 Units on a scale Standard Deviation 9.90	-2.2 Units on a scale Standard Deviation 10.91	—	0.6 Units on a scale Standard Deviation 10.85
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 6	-0.1 Units on a scale Standard Deviation 8.40	1.5 Units on a scale Standard Deviation 7.91	-1.2 Units on a scale Standard Deviation 8.59	7.5 Units on a scale Standard Deviation 14.85	0.0 Units on a scale Standard Deviation 8.46
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 12	-2.6 Units on a scale Standard Deviation 12.95	1.9 Units on a scale Standard Deviation 7.67	-5.5 Units on a scale Standard Deviation 14.75	-2.8 Units on a scale Standard Deviation 17.02	-2.6 Units on a scale Standard Deviation 12.99
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 18	-1.4 Units on a scale Standard Deviation 10.89	1.6 Units on a scale Standard Deviation 8.38	-4.1 Units on a scale Standard Deviation 12.12	—	-1.4 Units on a scale Standard Deviation 10.89
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 24	-5.4 Units on a scale Standard Deviation 13.47	-0.5 Units on a scale Standard Deviation 8.69	-9.8 Units on a scale Standard Deviation 15.48	—	-5.4 Units on a scale Standard Deviation 13.47
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 30	-5.6 Units on a scale Standard Deviation 15.02	1.0 Units on a scale Standard Deviation 6.23	-11.0 Units on a scale Standard Deviation 17.83	—	-5.6 Units on a scale Standard Deviation 15.02
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 6	-0.2 Units on a scale Standard Deviation 13.24	1.6 Units on a scale Standard Deviation 13.79	-1.5 Units on a scale Standard Deviation 12.75	1.0 Units on a scale Standard Deviation 7.07	-0.2 Units on a scale Standard Deviation 13.19
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 24	-7.0 Units on a scale Standard Deviation 16.69	-1.1 Units on a scale Standard Deviation 14.61	-12.4 Units on a scale Standard Deviation 16.74	—	-7.0 Units on a scale Standard Deviation 16.69
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 30	-10.5 Units on a scale Standard Deviation 19.64	1.1 Units on a scale Standard Deviation 10.29	-19.9 Units on a scale Standard Deviation 20.49	—	-10.5 Units on a scale Standard Deviation 19.64
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 6	0.5 Units on a scale Standard Deviation 18.13	-0.3 Units on a scale Standard Deviation 16.88	1.0 Units on a scale Standard Deviation 19.01	5.5 Units on a scale Standard Deviation 7.78	0.5 Units on a scale Standard Deviation 18.06
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 12	-3.6 Units on a scale Standard Deviation 20.09	-1.4 Units on a scale Standard Deviation 16.72	-5.0 Units on a scale Standard Deviation 21.95	-6.8 Units on a scale Standard Deviation 14.20	-3.7 Units on a scale Standard Deviation 19.96
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 18	-2.9 Units on a scale Standard Deviation 19.69	-2.1 Units on a scale Standard Deviation 19.31	-3.7 Units on a scale Standard Deviation 20.12	—	-2.9 Units on a scale Standard Deviation 19.69
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 24	-7.8 Units on a scale Standard Deviation 19.97	-7.1 Units on a scale Standard Deviation 18.46	-8.5 Units on a scale Standard Deviation 21.36	—	-7.8 Units on a scale Standard Deviation 19.97
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 30	-11.2 Units on a scale Standard Deviation 17.45	-6.4 Units on a scale Standard Deviation 13.80	-15.2 Units on a scale Standard Deviation 19.27	—	-11.2 Units on a scale Standard Deviation 17.45
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 6	0.1 Units on a scale Standard Deviation 18.37	0.2 Units on a scale Standard Deviation 15.69	0.0 Units on a scale Standard Deviation 20.09	-10.0 Units on a scale Standard Deviation 7.07	-0.0 Units on a scale Standard Deviation 18.32
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 12	-2.4 Units on a scale Standard Deviation 20.74	2.4 Units on a scale Standard Deviation 14.41	-5.6 Units on a scale Standard Deviation 23.49	-17.5 Units on a scale Standard Deviation 11.90	-2.7 Units on a scale Standard Deviation 20.69
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 18	-0.3 Units on a scale Standard Deviation 19.65	4.2 Units on a scale Standard Deviation 14.59	-4.2 Units on a scale Standard Deviation 22.57	—	-0.3 Units on a scale Standard Deviation 19.65
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 24	-8.5 Units on a scale Standard Deviation 20.69	-4.4 Units on a scale Standard Deviation 19.15	-12.3 Units on a scale Standard Deviation 21.44	—	-8.5 Units on a scale Standard Deviation 20.69
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 30	-4.9 Units on a scale Standard Deviation 19.54	4.9 Units on a scale Standard Deviation 12.73	-12.8 Units on a scale Standard Deviation 20.68	—	-4.9 Units on a scale Standard Deviation 19.54
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 6	0.2 Units on a scale Standard Deviation 8.39	1.0 Units on a scale Standard Deviation 7.99	-0.4 Units on a scale Standard Deviation 8.65	7.5 Units on a scale Standard Deviation 10.61	0.3 Units on a scale Standard Deviation 8.41
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 12	-2.6 Units on a scale Standard Deviation 11.36	1.0 Units on a scale Standard Deviation 9.04	-4.9 Units on a scale Standard Deviation 12.12	-5.5 Units on a scale Standard Deviation 6.40	-2.7 Units on a scale Standard Deviation 11.28
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 18	-1.6 Units on a scale Standard Deviation 10.10	1.1 Units on a scale Standard Deviation 9.54	-3.9 Units on a scale Standard Deviation 10.05	—	-1.6 Units on a scale Standard Deviation 10.10
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 24	-5.7 Units on a scale Standard Deviation 11.11	-2.3 Units on a scale Standard Deviation 10.32	-8.7 Units on a scale Standard Deviation 10.99	—	-5.7 Units on a scale Standard Deviation 11.11
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 12	-3.5 Units on a scale Standard Deviation 16.14	2.7 Units on a scale Standard Deviation 14.76	-7.5 Units on a scale Standard Deviation 15.78	-4.3 Units on a scale Standard Deviation 3.10	-3.5 Units on a scale Standard Deviation 15.98
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Global Functioning Scale and Each Core Scale Score (Pediatric Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 30	-6.7 Units on a scale Standard Deviation 12.37	-0.1 Units on a scale Standard Deviation 5.81	-12.0 Units on a scale Standard Deviation 13.73	—	-6.7 Units on a scale Standard Deviation 12.37

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies all participants in the Full Analysis Set with age between 11-18 years contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Outcome measures

Outcome measures
Measure	Total Participants n=58 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=31 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=27 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 6	-4.3 Units on a scale Standard Deviation 10.32	-2.1 Units on a scale Standard Deviation 8.82	-18.8 Units on a scale Standard Deviation 8.42	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 12	-3.5 Units on a scale Standard Deviation 12.57	-4.4 Units on a scale Standard Deviation 9.24	-2.4 Units on a scale Standard Deviation 16.17	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 18	-5.1 Units on a scale Standard Deviation 8.72	-6.4 Units on a scale Standard Deviation 8.34	1.3 Units on a scale Standard Deviation 9.24	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 24	-9.4 Units on a scale Standard Deviation 14.83	-11.0 Units on a scale Standard Deviation 13.47	-7.5 Units on a scale Standard Deviation 16.51	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 30	-7.6 Units on a scale Standard Deviation 9.71	-7.4 Units on a scale Standard Deviation 10.21	-8.0 Units on a scale Standard Deviation 11.31	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 6	-5.2 Units on a scale Standard Deviation 13.03	-5.1 Units on a scale Standard Deviation 13.87	-5.8 Units on a scale Standard Deviation 5.62	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 12	-12.2 Units on a scale Standard Deviation 15.73	-14.6 Units on a scale Standard Deviation 17.24	-9.2 Units on a scale Standard Deviation 13.31	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 18	-15.2 Units on a scale Standard Deviation 18.27	-19.3 Units on a scale Standard Deviation 15.26	5.7 Units on a scale Standard Deviation 20.65	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 24	-19.4 Units on a scale Standard Deviation 19.84	-27.1 Units on a scale Standard Deviation 21.79	-10.3 Units on a scale Standard Deviation 12.68	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 30	-22.4 Units on a scale Standard Deviation 25.42	-28.4 Units on a scale Standard Deviation 25.83	-1.5 Units on a scale Standard Deviation 6.36	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 6	-5.5 Units on a scale Standard Deviation 13.52	-5.9 Units on a scale Standard Deviation 14.24	-3.0 Units on a scale Standard Deviation 7.87	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 12	-9.4 Units on a scale Standard Deviation 14.07	-12.4 Units on a scale Standard Deviation 15.44	-5.4 Units on a scale Standard Deviation 11.21	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 18	-16.4 Units on a scale Standard Deviation 16.96	-18.8 Units on a scale Standard Deviation 17.62	-4.7 Units on a scale Standard Deviation 4.73	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 24	-14.1 Units on a scale Standard Deviation 17.69	-23.8 Units on a scale Standard Deviation 17.52	-2.6 Units on a scale Standard Deviation 8.99	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 6	-6.3 Units on a scale Standard Deviation 15.57	-7.2 Units on a scale Standard Deviation 14.64	-0.5 Units on a scale Standard Deviation 22.65	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 30	-18.1 Units on a scale Standard Deviation 24.92	-25.9 Units on a scale Standard Deviation 22.04	9.0 Units on a scale Standard Deviation 12.73	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 12	-9.2 Units on a scale Standard Deviation 22.54	-17.4 Units on a scale Standard Deviation 19.43	1.6 Units on a scale Standard Deviation 22.24	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 18	-14.4 Units on a scale Standard Deviation 16.88	-15.3 Units on a scale Standard Deviation 16.31	-10.0 Units on a scale Standard Deviation 22.91	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 24	-13.0 Units on a scale Standard Deviation 24.79	-22.3 Units on a scale Standard Deviation 20.23	-2.1 Units on a scale Standard Deviation 25.74	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 30	-17.8 Units on a scale Standard Deviation 22.79	-17.9 Units on a scale Standard Deviation 24.30	-17.5 Units on a scale Standard Deviation 24.75	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 6	-5.3 Units on a scale Standard Deviation 8.18	-5.0 Units on a scale Standard Deviation 8.34	-7.3 Units on a scale Standard Deviation 7.76	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 18	-10.4 Units on a scale Standard Deviation 8.77	-12.1 Units on a scale Standard Deviation 8.56	-2.0 Units on a scale Standard Deviation 3.61	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 24	-12.0 Units on a scale Standard Deviation 14.48	-19.0 Units on a scale Standard Deviation 12.31	-3.8 Units on a scale Standard Deviation 12.63	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 12	-6.4 Units on a scale Standard Deviation 11.32	-10.0 Units on a scale Standard Deviation 9.34	-1.5 Units on a scale Standard Deviation 12.10	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Sports, Month 30	-21.3 Units on a scale Standard Deviation 17.42	-24.7 Units on a scale Standard Deviation 17.74	-9.5 Units on a scale Standard Deviation 13.44	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 12	-0.3 Units on a scale Standard Deviation 29.07	-9.0 Units on a scale Standard Deviation 20.31	11.3 Units on a scale Standard Deviation 34.97	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 18	-5.2 Units on a scale Standard Deviation 19.03	-4.1 Units on a scale Standard Deviation 20.30	-10.3 Units on a scale Standard Deviation 12.01	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Pain, Month 24	-5.4 Units on a scale Standard Deviation 26.52	-14.2 Units on a scale Standard Deviation 20.41	4.9 Units on a scale Standard Deviation 29.67	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 6	-10.5 Units on a scale Standard Deviation 22.71	-13.1 Units on a scale Standard Deviation 19.57	7.5 Units on a scale Standard Deviation 36.63	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Parent Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 30	-17.4 Units on a scale Standard Deviation 16.40	-21.7 Units on a scale Standard Deviation 16.19	-2.5 Units on a scale Standard Deviation 2.12	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 6, 12, 18, 24 and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies all participants in the Full Analysis Set with age between 11-18 years contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Outcome measures

Outcome measures
Measure	Total Participants n=58 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=31 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=27 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 6	-1.5 Units on a scale Standard Deviation 8.03	-1.6 Units on a scale Standard Deviation 8.15	-1.0 Units on a scale Standard Deviation 8.29	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 18	-0.7 Units on a scale Standard Deviation 8.13	0.3 Units on a scale Standard Deviation 7.14	-5.7 Units on a scale Standard Deviation 12.70	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 24	-5.4 Units on a scale Standard Deviation 11.30	-5.3 Units on a scale Standard Deviation 10.93	-5.5 Units on a scale Standard Deviation 12.06	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 30	-4.7 Units on a scale Standard Deviation 6.46	-5.4 Units on a scale Standard Deviation 7.16	-2.0 Units on a scale Standard Deviation 2.83	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 18	-9.6 Units on a scale Standard Deviation 10.78	-9.6 Units on a scale Standard Deviation 11.78	-9.7 Units on a scale Standard Deviation 4.04	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 24	-17.1 Units on a scale Standard Deviation 19.86	-24.9 Units on a scale Standard Deviation 19.56	-7.9 Units on a scale Standard Deviation 16.34	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 30	-15.4 Units on a scale Standard Deviation 17.61	-17.0 Units on a scale Standard Deviation 19.71	-10.0 Units on a scale Standard Deviation 8.49	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Sports, Month 6	-1.2 Units on a scale Standard Deviation 13.56	-0.9 Units on a scale Standard Deviation 14.11	-2.8 Units on a scale Standard Deviation 10.40	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Sports, Month 12	-4.0 Units on a scale Standard Deviation 16.30	-4.6 Units on a scale Standard Deviation 19.60	-3.2 Units on a scale Standard Deviation 11.03	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Sports, Month 18	-7.7 Units on a scale Standard Deviation 13.52	-8.7 Units on a scale Standard Deviation 14.39	-2.7 Units on a scale Standard Deviation 7.77	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Sports, Month 24	-15.3 Units on a scale Standard Deviation 17.87	-21.4 Units on a scale Standard Deviation 19.71	-8.2 Units on a scale Standard Deviation 12.58	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Pain, Month 6	-1.6 Units on a scale Standard Deviation 17.34	-2.7 Units on a scale Standard Deviation 17.96	5.5 Units on a scale Standard Deviation 11.47	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Pain, Month 12	4.8 Units on a scale Standard Deviation 23.77	2.1 Units on a scale Standard Deviation 26.07	8.3 Units on a scale Standard Deviation 20.53	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Pain, Month 18	-3.4 Units on a scale Standard Deviation 18.30	-2.6 Units on a scale Standard Deviation 17.19	-7.7 Units on a scale Standard Deviation 27.32	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Pain, Month 24	-4.8 Units on a scale Standard Deviation 23.78	-12.4 Units on a scale Standard Deviation 24.21	4.0 Units on a scale Standard Deviation 20.55	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Pain, Month 30	10.8 Units on a scale Standard Deviation 20.92	13.9 Units on a scale Standard Deviation 23.10	0.0 Units on a scale Standard Deviation 0.00	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 6	-7.9 Units on a scale Standard Deviation 21.71	-7.8 Units on a scale Standard Deviation 23.14	-8.8 Units on a scale Standard Deviation 8.54	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 12	-1.6 Units on a scale Standard Deviation 20.48	-4.8 Units on a scale Standard Deviation 23.34	2.6 Units on a scale Standard Deviation 15.58	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 24	-5.9 Units on a scale Standard Deviation 26.53	-2.0 Units on a scale Standard Deviation 31.47	-10.6 Units on a scale Standard Deviation 19.11	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 12	-2.9 Units on a scale Standard Deviation 10.98	-4.4 Units on a scale Standard Deviation 12.66	-0.9 Units on a scale Standard Deviation 8.18	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Sports, Month 30	-8.4 Units on a scale Standard Deviation 12.32	-10.4 Units on a scale Standard Deviation 13.45	-1.5 Units on a scale Standard Deviation 2.12	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 18	-4.2 Units on a scale Standard Deviation 16.02	-3.3 Units on a scale Standard Deviation 17.29	-8.3 Units on a scale Standard Deviation 7.64	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Happiness, Month 30	-2.8 Units on a scale Standard Deviation 14.60	-2.9 Units on a scale Standard Deviation 16.29	-2.5 Units on a scale Standard Deviation 10.61	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 6	-2.1 Units on a scale Standard Deviation 6.82	-2.4 Units on a scale Standard Deviation 6.62	0.3 Units on a scale Standard Deviation 8.77	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 18	-5.2 Units on a scale Standard Deviation 7.72	-5.0 Units on a scale Standard Deviation 7.52	-6.3 Units on a scale Standard Deviation 10.41	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 24	-10.5 Units on a scale Standard Deviation 12.07	-15.9 Units on a scale Standard Deviation 12.85	-4.2 Units on a scale Standard Deviation 7.37	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Global Function, Month 30	-4.4 Units on a scale Standard Deviation 10.81	-4.7 Units on a scale Standard Deviation 12.46	-3.5 Units on a scale Standard Deviation 0.71	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Upper Extremity, Month 12	-4.0 Units on a scale Standard Deviation 11.58	-4.6 Units on a scale Standard Deviation 10.10	-3.3 Units on a scale Standard Deviation 13.53	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 6	-4.2 Units on a scale Standard Deviation 11.53	-4.5 Units on a scale Standard Deviation 10.71	-2.0 Units on a scale Standard Deviation 18.09	—	—
Change From Baseline in PODCI Global Functioning Scale and Each Core Scale Score (Adolescent Self Report) at Months 6, 12, 18, 24 and 30 Transfer and Mobility, Month 12	-8.8 Units on a scale Standard Deviation 17.38	-10.6 Units on a scale Standard Deviation 18.20	-6.3 Units on a scale Standard Deviation 16.40	—	—

SECONDARY outcome

Timeframe: Months 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure. 'Number Analyzed' signifies participants evaluable for specified rows. No ambulatory participants were \>=16 years hence N=0 in arms "Ambulatory participants with aged \>=6 years" and Total Ambulatory Participants".

EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The responses record three levels of severity (no problems/some or moderate problems/extreme problems) within a particular EQ-5D dimension. EQ-5D-3L assessment was only performed in participants \>= 16 years old as pre-specified in protocol.

Outcome measures

Outcome measures
Measure	Total Participants n=6 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=6 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Mobility, no problems, Month 12	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Mobility, some problems, Month 12	3 Participants	—	—	3 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Mobility, extreme problems, Month 12	3 Participants	—	—	3 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Self-Care, some problems, Month 12	4 Participants	—	—	4 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Pain/Discomfort, some problems, Month 12	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Self-Care, no problems, Month 12	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Self-Care, extreme problems, Month 12	2 Participants	—	—	2 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Usual Activities, no problems, Month 12	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Usual Activities, some problems, Month 12	3 Participants	—	—	3 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Usual Activities, extreme problems, Month 12	2 Participants	—	—	2 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Pain/Discomfort, no problems, Month 12	5 Participants	—	—	5 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Pain/Discomfort, extreme problems, Month 12	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Anxiety/Depression, no problems, Month 12	5 Participants	—	—	5 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Anxiety/Depression, some problems, Month 12	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each European Quality of Life (EuroQoL) 5 Dimension 3 Level (EQ-5D-3L) 5 Dimensions at Month 12 Anxiety/Depression, extreme problems, Month 12	0 Participants	—	—	0 Participants	—

SECONDARY outcome

Timeframe: Month 24

Outcome measures

Outcome measures
Measure	Total Participants n=8 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=2 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=2 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=10 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Self-Care, no problems, Month 24	0 Participants	0 Participants	0 Participants	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Self-Care, some problems, Month 24	3 Participants	1 Participants	1 Participants	4 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Usual Activities, extreme problems, Month 24	3 Participants	1 Participants	1 Participants	4 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Pain/Discomfort, no problems, Month 24	5 Participants	1 Participants	1 Participants	6 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Pain/Discomfort, extreme problems, Month 24	0 Participants	0 Participants	0 Participants	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Anxiety/Depression, no problems, Month 24	7 Participants	1 Participants	1 Participants	8 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Anxiety/Depression, extreme problems, Month 24	0 Participants	0 Participants	0 Participants	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Pain/Discomfort, some problems, Month 24	3 Participants	1 Participants	1 Participants	4 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Anxiety/Depression, some problems, Month 24	1 Participants	1 Participants	1 Participants	2 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Mobility, no problems, Month 24	0 Participants	1 Participants	1 Participants	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Mobility, some problems, Month 24	4 Participants	0 Participants	0 Participants	4 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Mobility, extreme problems, Month 24	4 Participants	1 Participants	1 Participants	5 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Self-Care, extreme problems, Month 24	5 Participants	1 Participants	1 Participants	6 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Usual Activities, no problems, Month 24	0 Participants	1 Participants	1 Participants	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 24 Usual Activities, some problems, Month 24	5 Participants	0 Participants	0 Participants	5 Participants	—

SECONDARY outcome

Timeframe: Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure. No participants were available for analysis at Month 30 in arms 'ambulatory participants with age \>=6 years' and 'total ambulatory participants' hence, 'Number of Participants Analyzed' and 'Number Analyzed' were kept 0 for these arms.

Outcome measures

Outcome measures
Measure	Total Participants n=1 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=1 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Mobility, no problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Mobility, some problems, Month 30	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Mobility, extreme problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Self-Care, no problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Usual Activities, no problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Anxiety/Depression, no problems, Month 30	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Anxiety/Depression, some problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Anxiety/Depression, extreme problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Self-Care, some problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Self-Care, extreme problems, Month 30	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Usual Activities, some problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Usual Activities, extreme problems, Month 30	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Pain/Discomfort, no problems, Month 30	1 Participants	—	—	1 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Pain/Discomfort, some problems, Month 30	0 Participants	—	—	0 Participants	—
Number of Participants According to Response to Each EQ-5D-3L 5 Dimensions at Month 30 Pain/Discomfort, extreme problems, Month 30	0 Participants	—	—	0 Participants	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24 and 30

EQ-5D-3L is a participant completed questionnaire designed to assess impact on health related quality of life. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state), where higher scores indicated better quality of life. EQ-VAS assessment was only performed in the participants \>= 16 years old.

Outcome measures

Outcome measures
Measure	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=4 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in EQ-5D-3L Visual Analogue Score (VAS) Assessment at Months 12, 24 and 30 Month 12	—	-4.0 Units on a scale Standard Deviation 9.54	-4.0 Units on a scale Standard Deviation 9.54	—	—
Change From Baseline in EQ-5D-3L Visual Analogue Score (VAS) Assessment at Months 12, 24 and 30 Month 24	—	-11.3 Units on a scale Standard Deviation 7.37	-11.3 Units on a scale Standard Deviation 7.37	—	—
Change From Baseline in EQ-5D-3L Visual Analogue Score (VAS) Assessment at Months 12, 24 and 30 Month 30	—	-14.0 Units on a scale Standard Deviation NA Standard deviation could not be calculated for single participant.	-14.0 Units on a scale Standard Deviation NA Standard deviation could not be calculated for single participant.	—	—

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

EQ-5D-3L index score, participants rated their current health state on 5 single-item dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression with each dimension having three levels of severity: 1=no problems, 2=some problems and 3=extreme problems. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total index score range of 0 to 1.00. Higher scores indicating a better quality of life. EQ-5D-3L was only performed in participants \>= 16 years old.

Outcome measures

Outcome measures
Measure	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=4 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=4 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in EQ-5D-3L Index Score at Months 12, 24 and 30 Month 30	—	-0.3 Units on a scale Standard Deviation NA Standard deviation could not be calculated for the single participant.	-0.3 Units on a scale Standard Deviation NA Standard deviation could not be calculated for the single participant.	—	—
Change From Baseline in EQ-5D-3L Index Score at Months 12, 24 and 30 Month 12	—	-0.0 Units on a scale Standard Deviation 0.14	-0.0 Units on a scale Standard Deviation 0.14	—	—
Change From Baseline in EQ-5D-3L Index Score at Months 12, 24 and 30 Month 24	—	-0.2 Units on a scale Standard Deviation 0.16	-0.2 Units on a scale Standard Deviation 0.16	—	—

SECONDARY outcome

Timeframe: Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. All participants reported under "Number of Participants Analyzed" contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems.

Outcome measures

Outcome measures
Measure	Total Participants n=217 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=57 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=160 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=24 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=241 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Mobility, no problems, Month 12	136 Participants	45 Participants	91 Participants	0 Participants	136 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Self-Care, no problems, Month 12	92 Participants	22 Participants	70 Participants	3 Participants	95 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Pain/Discomfort, some problems, Month 12	78 Participants	21 Participants	57 Participants	7 Participants	85 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Mobility, some problems, Month 12	64 Participants	12 Participants	52 Participants	0 Participants	64 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Mobility, extreme problems, Month 12	17 Participants	0 Participants	17 Participants	24 Participants	41 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Self-Care, some problems, Month 12	98 Participants	25 Participants	73 Participants	7 Participants	105 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Self-Care, extreme problems, Month 12	27 Participants	10 Participants	17 Participants	14 Participants	41 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Usual Activities, no problems, Month 12	110 Participants	40 Participants	70 Participants	4 Participants	114 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Usual Activities, some problems, Month 12	91 Participants	17 Participants	74 Participants	6 Participants	97 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Usual Activities, extreme problems, Month 12	16 Participants	0 Participants	16 Participants	14 Participants	30 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Pain/Discomfort, no problems, Month 12	137 Participants	36 Participants	101 Participants	17 Participants	154 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Pain/Discomfort, extreme problems, Month 12	2 Participants	0 Participants	2 Participants	0 Participants	2 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Anxiety/Depression, no problems, Month 12	155 Participants	45 Participants	110 Participants	14 Participants	169 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Anxiety/Depression, some problems, Month 12	49 Participants	8 Participants	41 Participants	9 Participants	58 Participants
Number of Participants According to Response to Each EuroQoL 5 Dimension Youth (EQ-5D-Y) 5 Dimensions at Month 12 Anxiety/Depression, extreme problems, Month 12	13 Participants	4 Participants	9 Participants	1 Participants	14 Participants

SECONDARY outcome

Timeframe: Month 24

Outcome measures

Outcome measures
Measure	Total Participants n=192 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=58 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=134 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=15 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=207 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Mobility, no problems, Month 24	104 Participants	48 Participants	56 Participants	0 Participants	104 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Mobility, some problems, Month 24	65 Participants	10 Participants	55 Participants	0 Participants	65 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Mobility, extreme problems, Month 24	23 Participants	0 Participants	23 Participants	15 Participants	38 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Self-Care, no problems, Month 24	97 Participants	32 Participants	65 Participants	1 Participants	98 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Self-Care, some problems, Month 24	74 Participants	21 Participants	53 Participants	5 Participants	79 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Self-Care, extreme problems, Month 24	21 Participants	5 Participants	16 Participants	9 Participants	30 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Usual Activities, no problems, Month 24	91 Participants	42 Participants	49 Participants	6 Participants	97 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Usual Activities, some problems, Month 24	75 Participants	15 Participants	60 Participants	3 Participants	78 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Usual Activities, extreme problems, Month 24	26 Participants	1 Participants	25 Participants	6 Participants	32 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Pain/Discomfort, no problems, Month 24	118 Participants	38 Participants	80 Participants	12 Participants	130 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Pain/Discomfort, some problems, Month 24	71 Participants	19 Participants	52 Participants	3 Participants	74 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Pain/Discomfort, extreme problems, Month 24	3 Participants	1 Participants	2 Participants	0 Participants	3 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Anxiety/Depression, no problems, Month 24	134 Participants	43 Participants	91 Participants	11 Participants	145 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Anxiety/Depression, some problems, Month 24	55 Participants	14 Participants	41 Participants	3 Participants	58 Participants
Number of Participants According to Response to Each EQ-5D-Y 5 Dimensions at Month 24 Anxiety/Depression, extreme problems, Month 24	3 Participants	1 Participants	2 Participants	1 Participants	4 Participants

SECONDARY outcome

Timeframe: Month 30

Outcome measures

Outcome measures
Measure	Total Participants n=104 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=26 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=78 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=3 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=107 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Self-Care, some problems, Month 30	31 Participants	8 Participants	23 Participants	2 Participants	33 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Self-Care, extreme problems, Month 30	12 Participants	2 Participants	10 Participants	1 Participants	13 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Usual Activities, some problems, Month 30	50 Participants	10 Participants	40 Participants	0 Participants	50 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Usual Activities, no problems, Month 30	44 Participants	16 Participants	28 Participants	3 Participants	47 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Mobility, no problems, Month 30	60 Participants	26 Participants	34 Participants	0 Participants	60 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Mobility, some problems, Month 30	34 Participants	0 Participants	34 Participants	0 Participants	34 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Mobility, extreme problems, Month 30	10 Participants	0 Participants	10 Participants	3 Participants	13 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Self-Care, no problems, Month 30	61 Participants	16 Participants	45 Participants	0 Participants	61 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Usual Activities, extreme problems, Month 30	10 Participants	0 Participants	10 Participants	0 Participants	10 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Pain/Discomfort, no problems, Month 30	61 Participants	17 Participants	44 Participants	3 Participants	64 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Pain/Discomfort, some problems, Month 30	39 Participants	9 Participants	30 Participants	0 Participants	39 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Pain/Discomfort, extreme problems, Month 30	4 Participants	0 Participants	4 Participants	0 Participants	4 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Anxiety/Depression, no problems, Month 30	66 Participants	18 Participants	48 Participants	1 Participants	67 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Anxiety/Depression, some problems, Month 30	34 Participants	8 Participants	26 Participants	1 Participants	35 Participants
Number of Participants According to Response to EQ-5D-Y 5 Dimensions at Month 30 Anxiety/Depression, extreme problems, Month 30	4 Participants	0 Participants	4 Participants	1 Participants	5 Participants

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set with age \<16 years and contributed data to table, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<16 years old. EQ-VAS recorded the participant's self-rated health on a vertical scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=32 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=308 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in EQ-5D-Y VAS Assessment at Months 12, 24, and 30 Month 12	-3.5 Units on a scale Standard Deviation 22.36	-1.0 Units on a scale Standard Deviation 19.84	-4.3 Units on a scale Standard Deviation 23.02	-5.0 Units on a scale Standard Deviation 22.94	-3.7 Units on a scale Standard Deviation 22.37
Change From Baseline in EQ-5D-Y VAS Assessment at Months 12, 24, and 30 Month 24	-5.0 Units on a scale Standard Deviation 20.19	-3.0 Units on a scale Standard Deviation 15.08	-5.6 Units on a scale Standard Deviation 21.59	-5.9 Units on a scale Standard Deviation 14.59	-5.1 Units on a scale Standard Deviation 19.78
Change From Baseline in EQ-5D-Y VAS Assessment at Months 12, 24, and 30 Month 30	-5.1 Units on a scale Standard Deviation 20.30	-1.0 Units on a scale Standard Deviation 12.16	-6.1 Units on a scale Standard Deviation 21.71	5.0 Units on a scale Standard Deviation 5.00	-4.8 Units on a scale Standard Deviation 20.07

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set with age \<16 years and contributed data to table, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

EQ-5D-Y is participant completed questionnaire designed to assess impact on health related quality of life in children and adolescents \<=16 years old. It is comprised of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: 1=no problems, 2=some problems, and 3=extreme problems. Participants indicated their health state by choosing the appropriate level from each dimension. The 5 digit health states thus obtained for each dimension were then converted into a single median index value. A health state index score was calculated from individual health profiles using a USA scoring algorithm. Health state index scores generally ranged from -0.109 to 1, where, -0.109= the worst health status, 1= full health. Higher the score the better the better quality of life.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=32 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=308 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in EQ-5D-Y Index Score at Months 12, 24, and 30 Month 12	-0.0 Units on a scale Standard Deviation 0.15	-0.0 Units on a scale Standard Deviation 0.12	-0.0 Units on a scale Standard Deviation 0.16	-0.0 Units on a scale Standard Deviation 0.13	-0.0 Units on a scale Standard Deviation 0.15
Change From Baseline in EQ-5D-Y Index Score at Months 12, 24, and 30 Month 24	-0.1 Units on a scale Standard Deviation 0.16	0.0 Units on a scale Standard Deviation 0.13	-0.1 Units on a scale Standard Deviation 0.16	-0.0 Units on a scale Standard Deviation 0.12	-0.1 Units on a scale Standard Deviation 0.16
Change From Baseline in EQ-5D-Y Index Score at Months 12, 24, and 30 Month 30	-0.1 Units on a scale Standard Deviation 0.17	0.0 Units on a scale Standard Deviation 0.09	-0.1 Units on a scale Standard Deviation 0.18	0.0 Units on a scale Standard Deviation 0.09	-0.1 Units on a scale Standard Deviation 0.17

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Number of Participants Analyzed' signifies all participants in the Full Analysis Set. All participants reported under "Number of Participants Analyzed" contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to their child's use of healthcare professionals, emergency room visits, and hospitalizations in past 3 months. Change from baseline in mean number of visits to primary care physician, emergency room and office visits is presented in this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To Primary Care Physician at Month 12	-0.0 Visits Standard Deviation 0.37	-0.1 Visits Standard Deviation 0.27	-0.0 Visits Standard Deviation 0.41	-0.0 Visits Standard Deviation 0.18	-0.0 Visits Standard Deviation 0.36
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To Primary Care Physician at Month 24	-0.0 Visits Standard Deviation 0.35	0.0 Visits Standard Deviation 0.23	-0.0 Visits Standard Deviation 0.41	0.1 Visits Standard Deviation 0.51	-0.0 Visits Standard Deviation 0.37
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To Primary Care Physician at Month 30	-0.0 Visits Standard Deviation 0.53	-0.1 Visits Standard Deviation 0.43	0.0 Visits Standard Deviation 0.55	0.0 Visits Standard Deviation 0.00	-0.0 Visits Standard Deviation 0.52
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To General Practitioner at Month 12	-0.1 Visits Standard Deviation 0.42	-0.1 Visits Standard Deviation 0.45	-0.0 Visits Standard Deviation 0.40	-0.1 Visits Standard Deviation 0.35	-0.1 Visits Standard Deviation 0.41
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To General Practitioner at Month 24	-0.0 Visits Standard Deviation 0.44	0.0 Visits Standard Deviation 0.57	-0.1 Visits Standard Deviation 0.36	-0.2 Visits Standard Deviation 0.52	-0.1 Visits Standard Deviation 0.45
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To General Practitioner at Month 30	-0.1 Visits Standard Deviation 0.43	-0.1 Visits Standard Deviation 0.40	-0.1 Visits Standard Deviation 0.44	0.0 Visits Standard Deviation 0.00	-0.1 Visits Standard Deviation 0.42
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To ER Due to Disease at Month 12	-0.0 Visits Standard Deviation 0.23	-0.0 Visits Standard Deviation 0.11	0.0 Visits Standard Deviation 0.27	0.0 Visits Standard Deviation 0.00	-0.0 Visits Standard Deviation 0.22
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To ER Due to Disease at Month 24	0.0 Visits Standard Deviation 0.10	-0.0 Visits Standard Deviation 0.12	0.0 Visits Standard Deviation 0.10	0.0 Visits Standard Deviation 0.00	0.0 Visits Standard Deviation 0.09
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 To ER Due to Disease at Month 30	-0.0 Visits Standard Deviation 0.19	-0.0 Visits Standard Deviation 0.18	-0.0 Visits Standard Deviation 0.20	0.0 Visits Standard Deviation 0.00	-0.0 Visits Standard Deviation 0.19
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 Office Visits at Month 12	-0.2 Visits Standard Deviation 6.18	-1.6 Visits Standard Deviation 7.04	0.5 Visits Standard Deviation 5.58	-3.4 Visits Standard Deviation 11.20	-0.6 Visits Standard Deviation 6.96
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 Office Visits at Month 24	0.2 Visits Standard Deviation 7.48	-0.4 Visits Standard Deviation 9.09	0.5 Visits Standard Deviation 6.44	-6.2 Visits Standard Deviation 15.70	-0.5 Visits Standard Deviation 8.78
Change From Baseline in Healthcare Resource Utilization (HRU) Survey Responses: Visit to Primary Care Physician, Emergency Room and Office Visits at Months 12, 24, and 30 Office Visits at Month 30	-0.7 Visits Standard Deviation 9.32	-2.0 Visits Standard Deviation 12.84	-0.2 Visits Standard Deviation 7.60	-17.0 Visits Standard Deviation 32.67	-1.3 Visits Standard Deviation 11.04

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

HRU questionnaire is completed by the caregiver and had questions about healthcare resources utilization related to number of nights in hospital due to disease/dedication for disease in past 3 months. Change from baseline in mean number of nights in hospital due to disease or medication for disease is presented in this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in HRU Survey Responses: Number of Nights in Hospital Due to Disease/Medication for Disease at Months 12, 24, and 30 Month 12	-0.2 Nights Standard Deviation 1.60	-0.2 Nights Standard Deviation 1.02	-0.2 Nights Standard Deviation 1.82	0.0 Nights Standard Deviation 0.00	-0.2 Nights Standard Deviation 1.51
Change From Baseline in HRU Survey Responses: Number of Nights in Hospital Due to Disease/Medication for Disease at Months 12, 24, and 30 Month 24	-0.2 Nights Standard Deviation 1.70	-0.2 Nights Standard Deviation 1.06	-0.2 Nights Standard Deviation 1.97	0.2 Nights Standard Deviation 0.83	-0.2 Nights Standard Deviation 1.64
Change From Baseline in HRU Survey Responses: Number of Nights in Hospital Due to Disease/Medication for Disease at Months 12, 24, and 30 Month 30	-0.1 Nights Standard Deviation 0.67	0.0 Nights Standard Deviation 0.00	-0.1 Nights Standard Deviation 0.79	0.0 Nights Standard Deviation 0.00	-0.1 Nights Standard Deviation 0.66

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Caregivers were asked to estimate out-of-pocket costs related to healthcare resource utilization. The number of out of pocket money was defined as the total spent of the past three months in managing child's Duchenne muscular dystrophy.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Out-of-Pocket Money of HRU Survey at Months 12, 24 and 30 Month 30	378.0 Chinese Yuan (CYN) Standard Deviation 6909.59	-1794.5 Chinese Yuan (CYN) Standard Deviation 6772.80	1213.6 Chinese Yuan (CYN) Standard Deviation 6820.65	-675.0 Chinese Yuan (CYN) Standard Deviation 4592.29	340.4 Chinese Yuan (CYN) Standard Deviation 6828.66
Change From Baseline in Out-of-Pocket Money of HRU Survey at Months 12, 24 and 30 Month 12	-598.7 Chinese Yuan (CYN) Standard Deviation 9713.33	-2748.1 Chinese Yuan (CYN) Standard Deviation 6100.61	476.0 Chinese Yuan (CYN) Standard Deviation 10947.21	1836.7 Chinese Yuan (CYN) Standard Deviation 6410.95	-328.1 Chinese Yuan (CYN) Standard Deviation 9425.78
Change From Baseline in Out-of-Pocket Money of HRU Survey at Months 12, 24 and 30 Month 24	627.3 Chinese Yuan (CYN) Standard Deviation 7925.23	352.8 Chinese Yuan (CYN) Standard Deviation 8484.07	777.6 Chinese Yuan (CYN) Standard Deviation 7630.02	2326.1 Chinese Yuan (CYN) Standard Deviation 6126.93	793.6 Chinese Yuan (CYN) Standard Deviation 7773.09

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

WPAI:CG is a self-reported measure of work productivity and impairment, was completed by caregiver, which had four scores: absenteeism (work time missed); presenteeism (impairment at work/reduced on the job effectiveness); work productivity loss (overall work impairment/absenteeism plus presenteeism); and activity impairment. Each score was expressed as a percentage (0-100%) with higher score indicating greater impairment and less productivity.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Activity Impairment as Per Work Productivity and Activity Impairment Questionnaire Adapted for Caregiving (WPAI:CG) Caregiver Unchanged at Months 12, 24, and 30 Month 12	2.0 Percentage activity impairment Standard Deviation 32.09	-6.6 Percentage activity impairment Standard Deviation 30.71	5.6 Percentage activity impairment Standard Deviation 32.08	-6.8 Percentage activity impairment Standard Deviation 34.37	0.9 Percentage activity impairment Standard Deviation 32.41
Change From Baseline in Percent Activity Impairment as Per Work Productivity and Activity Impairment Questionnaire Adapted for Caregiving (WPAI:CG) Caregiver Unchanged at Months 12, 24, and 30 Month 24	8.3 Percentage activity impairment Standard Deviation 33.76	7.4 Percentage activity impairment Standard Deviation 32.22	8.8 Percentage activity impairment Standard Deviation 34.68	-7.9 Percentage activity impairment Standard Deviation 31.02	6.6 Percentage activity impairment Standard Deviation 33.77
Change From Baseline in Percent Activity Impairment as Per Work Productivity and Activity Impairment Questionnaire Adapted for Caregiving (WPAI:CG) Caregiver Unchanged at Months 12, 24, and 30 Month 30	10.1 Percentage activity impairment Standard Deviation 31.65	-6.7 Percentage activity impairment Standard Deviation 31.14	13.8 Percentage activity impairment Standard Deviation 30.82	0.0 Percentage activity impairment Standard Deviation NA Standard deviation could not be calculated for single participant.	10.0 Percentage activity impairment Standard Deviation 31.43

SECONDARY outcome

Timeframe: Baseline (Day 1) and Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=93 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=35 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=58 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=11 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=104 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 12	-5.5 Percentage work time missed Standard Deviation 25.15	-6.1 Percentage work time missed Standard Deviation 25.47	-5.0 Percentage work time missed Standard Deviation 25.17	3.7 Percentage work time missed Standard Deviation 18.59	-4.5 Percentage work time missed Standard Deviation 24.63

SECONDARY outcome

Timeframe: Baseline (Day 1) and Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=74 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=33 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=41 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=11 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=85 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 24	1.0 Percentage work time missed Standard Deviation 26.91	1.1 Percentage work time missed Standard Deviation 24.46	0.9 Percentage work time missed Standard Deviation 29.04	-4.4 Percentage work time missed Standard Deviation 23.92	0.3 Percentage work time missed Standard Deviation 26.47

SECONDARY outcome

Timeframe: Baseline (Day 1) and Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. Here, "Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=28 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=9 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=19 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=1 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=29 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Work Time Missed as Per WPAI: CG Caregiver Unchanged at Month 30	-5.6 Percentage work time missed Standard Deviation 21.73	-21.5 Percentage work time missed Standard Deviation 26.90	2.0 Percentage work time missed Standard Deviation 14.15	0.0 Percentage work time missed Standard Deviation NA Standard deviation could not be calculated for single participant.	-5.4 Percentage work time missed Standard Deviation 21.36

SECONDARY outcome

Timeframe: Baseline (Day 1) and Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30 Percent Overall Work Impairment, Month 12	-0.4 Percentage work impairment Standard Deviation 25.83	-5.2 Percentage work impairment Standard Deviation 26.25	2.6 Percentage work impairment Standard Deviation 25.35	-9.3 Percentage work impairment Standard Deviation 36.17	-1.3 Percentage work impairment Standard Deviation 26.93
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30 Percent Overall Work Impairment, Month 24	2.7 Percentage work impairment Standard Deviation 27.16	0.2 Percentage work impairment Standard Deviation 27.83	4.8 Percentage work impairment Standard Deviation 26.78	-12.7 Percentage work impairment Standard Deviation 39.72	0.9 Percentage work impairment Standard Deviation 29.09
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30 Percent Overall Work Impairment, Month 30	0.8 Percentage work impairment Standard Deviation 28.94	-11.9 Percentage work impairment Standard Deviation 35.09	6.8 Percentage work impairment Standard Deviation 24.29	30.0 Percentage work impairment Standard Deviation NA Standard deviation could not be calculated for single participant.	1.8 Percentage work impairment Standard Deviation 28.93
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30 Percent impairment while working, Month 12	1.5 Percentage work impairment Standard Deviation 22.84	-3.7 Percentage work impairment Standard Deviation 19.87	4.7 Percentage work impairment Standard Deviation 24.08	-14.0 Percentage work impairment Standard Deviation 33.40	0.0 Percentage work impairment Standard Deviation 24.29
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30 Percent impairment while working, Month 24	1.2 Percentage work impairment Standard Deviation 26.15	-1.2 Percentage work impairment Standard Deviation 25.47	3.1 Percentage work impairment Standard Deviation 26.82	-15.0 Percentage work impairment Standard Deviation 37.49	-0.7 Percentage work impairment Standard Deviation 27.94
Change From Baseline in Percent Overall Work Impairment WPAI: CG Caregiver Unchanged at Months 12, 24, and 30 Percent impairment while working, Month 30	3.9 Percentage work impairment Standard Deviation 25.72	1.1 Percentage work impairment Standard Deviation 28.48	5.3 Percentage work impairment Standard Deviation 25.03	30.0 Percentage work impairment Standard Deviation NA Standard deviation could not be calculated for single participant.	4.8 Percentage work impairment Standard Deviation 25.72

SECONDARY outcome

Timeframe: Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Percent Activity Impairment Score as Per WPAI: CG at Months 12, 24, and 30 Month 12	42.5 Percentage activity impairment Standard Deviation 29.36	30.9 Percentage activity impairment Standard Deviation 26.63	48.3 Percentage activity impairment Standard Deviation 29.02	61.7 Percentage activity impairment Standard Deviation 31.63	44.6 Percentage activity impairment Standard Deviation 30.17
Percent Activity Impairment Score as Per WPAI: CG at Months 12, 24, and 30 Month 24	48.1 Percentage activity impairment Standard Deviation 28.85	41.3 Percentage activity impairment Standard Deviation 29.38	51.8 Percentage activity impairment Standard Deviation 27.98	62.6 Percentage activity impairment Standard Deviation 32.78	49.5 Percentage activity impairment Standard Deviation 29.50
Percent Activity Impairment Score as Per WPAI: CG at Months 12, 24, and 30 Month 30	47.1 Percentage activity impairment Standard Deviation 29.45	23.7 Percentage activity impairment Standard Deviation 23.41	56.2 Percentage activity impairment Standard Deviation 26.49	65.0 Percentage activity impairment Standard Deviation 31.09	47.8 Percentage activity impairment Standard Deviation 29.55

SECONDARY outcome

Timeframe: Month 12

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=147 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=58 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=89 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=16 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=163 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Percent Work Time Missed Score as Per WPAI: CG at Month 12	7.1 Percentage work time missed Standard Deviation 14.41	6.8 Percentage work time missed Standard Deviation 14.98	7.3 Percentage work time missed Standard Deviation 14.11	21.1 Percentage work time missed Standard Deviation 28.81	8.5 Percentage work time missed Standard Deviation 16.78

SECONDARY outcome

Timeframe: Month 24

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=131 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=60 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=71 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=15 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=146 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Percent Work Time Missed Score as Per WPAI: CG at Month 24	11.9 Percentage work time missed Standard Deviation 21.56	10.5 Percentage work time missed Standard Deviation 19.70	13.0 Percentage work time missed Standard Deviation 23.10	8.7 Percentage work time missed Standard Deviation 16.50	11.5 Percentage work time missed Standard Deviation 21.07

SECONDARY outcome

Timeframe: Month 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Total Participants n=67 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=27 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=40 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=3 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=70 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Percent Work Time Missed Score as Per WPAI: CG at Month 30	9.4 Percentage work time missed Standard Deviation 16.21	2.9 Percentage work time missed Standard Deviation 6.67	13.8 Percentage work time missed Standard Deviation 19.15	0.0 Percentage work time missed Standard Deviation 0.00	9.0 Percentage work time missed Standard Deviation 15.97

SECONDARY outcome

Timeframe: Months 12, 24, and 30

Population: Full Analysis Set included all participants who had been enrolled in the study. 'Number of Participants Analyzed' signifies all participants in the Full Analysis Set and contributed data to the table; however, may not have evaluable data for every row. 'Number Analyzed' signifies participants evaluable for specified rows.

Outcome measures

Outcome measures
Measure	Total Participants n=276 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.	Ambulatory Participants With Age >=6 Years n=99 Participants Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=177 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 Participants Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Total Participants n=312 Participants Participants diagnosed with DMD who were ambulant and who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained CE with the inability to perform the 10-meter run/walk assessment) were included.
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30 Percent impairment while working, Month 30	35.5 Percentage work impairment Standard Deviation 27.04	21.1 Percentage work impairment Standard Deviation 24.23	45.3 Percentage work impairment Standard Deviation 24.60	56.7 Percentage work impairment Standard Deviation 20.82	36.4 Percentage work impairment Standard Deviation 27.03
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30 Percent Overall Work Impairment, Month 12	39.9 Percentage work impairment Standard Deviation 26.60	32.0 Percentage work impairment Standard Deviation 25.70	45.1 Percentage work impairment Standard Deviation 26.4	46.6 Percentage work impairment Standard Deviation 29.38	40.5 Percentage work impairment Standard Deviation 26.85
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30 Percent Overall Work Impairment, Month 24	41.1 Percentage work impairment Standard Deviation 27.70	37.2 Percentage work impairment Standard Deviation 27.64	44.3 Percentage work impairment Standard Deviation 27.53	45.9 Percentage work impairment Standard Deviation 29.86	41.6 Percentage work impairment Standard Deviation 27.86
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30 Percent Overall Work Impairment, Month 30	40.4 Percentage work impairment Standard Deviation 28.63	23.3 Percentage work impairment Standard Deviation 24.41	51.9 Percentage work impairment Standard Deviation 25.52	56.7 Percentage work impairment Standard Deviation 20.82	41.1 Percentage work impairment Standard Deviation 28.42
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30 Percent impairment while working, Month 12	36.1 Percentage work impairment Standard Deviation 25.36	27.9 Percentage work impairment Standard Deviation 23.90	41.3 Percentage work impairment Standard Deviation 25.01	40.7 Percentage work impairment Standard Deviation 26.58	36.5 Percentage work impairment Standard Deviation 25.43
Percent Overall Work Impairment Scores as Per WPAI: CG at Months 12, 24, and 30 Percent impairment while working, Month 24	35.4 Percentage work impairment Standard Deviation 25.52	32.5 Percentage work impairment Standard Deviation 24.95	37.7 Percentage work impairment Standard Deviation 25.92	41.3 Percentage work impairment Standard Deviation 28.00	36.0 Percentage work impairment Standard Deviation 25.75

Adverse Events

Ambulatory Participants With Age <6 Years

Serious events: 0 serious events

Other events: 84 other events

Deaths: 1 deaths

Ambulatory Participants With Age >=6 Years

Serious events: 0 serious events

Other events: 136 other events

Deaths: 0 deaths

Non-ambulatory Participants

Serious events: 0 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ambulatory Participants With Age <6 Years n=99 participants at risk Participants diagnosed with DMD who were ambulant and were less than 6 years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the standard of care (SOC).	Ambulatory Participants With Age >=6 Years n=177 participants at risk Participants diagnosed with DMD who were ambulant and were greater than or equal to (\>=6) years of age at the time of screening were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.	Non-ambulatory Participants n=36 participants at risk Participants diagnosed with DMD who were non-ambulatory (i.e., used wheel chair full-time or were unable to complete daily activities \[such as going to bathroom\] by walking independently, based on performance reported at home by participant or caregiver and verification by trained clinical evaluator \[CE\] with the inability to perform the 10-meter run/walk assessment) were included. No investigational drug was administered, and participants were allowed to take medications prescribed by their physicians, which constituted the SOC.
Blood and lymphatic system disorders Leukocytosis	19.2% 19/99 • Up to Month 30	13.6% 24/177 • Up to Month 30	13.9% 5/36 • Up to Month 30
Cardiac disorders Sinus tachycardia	5.1% 5/99 • Up to Month 30	1.7% 3/177 • Up to Month 30	0.00% 0/36 • Up to Month 30
Gastrointestinal disorders Abdominal pain	5.1% 5/99 • Up to Month 30	1.1% 2/177 • Up to Month 30	2.8% 1/36 • Up to Month 30
Gastrointestinal disorders Constipation	1.0% 1/99 • Up to Month 30	0.56% 1/177 • Up to Month 30	5.6% 2/36 • Up to Month 30
Gastrointestinal disorders Vomiting	5.1% 5/99 • Up to Month 30	1.1% 2/177 • Up to Month 30	0.00% 0/36 • Up to Month 30
General disorders Pyrexia	9.1% 9/99 • Up to Month 30	4.0% 7/177 • Up to Month 30	2.8% 1/36 • Up to Month 30
Infections and infestations Bronchitis	12.1% 12/99 • Up to Month 30	2.3% 4/177 • Up to Month 30	0.00% 0/36 • Up to Month 30
Infections and infestations COVID-19	6.1% 6/99 • Up to Month 30	12.4% 22/177 • Up to Month 30	2.8% 1/36 • Up to Month 30
Infections and infestations Nasopharyngitis	4.0% 4/99 • Up to Month 30	5.6% 10/177 • Up to Month 30	5.6% 2/36 • Up to Month 30
Infections and infestations Pharyngitis	5.1% 5/99 • Up to Month 30	1.1% 2/177 • Up to Month 30	0.00% 0/36 • Up to Month 30
Infections and infestations Tonsillitis	5.1% 5/99 • Up to Month 30	2.8% 5/177 • Up to Month 30	0.00% 0/36 • Up to Month 30
Infections and infestations Upper respiratory tract infection	74.7% 74/99 • Up to Month 30	50.3% 89/177 • Up to Month 30	36.1% 13/36 • Up to Month 30
Injury, poisoning and procedural complications Fall	2.0% 2/99 • Up to Month 30	5.1% 9/177 • Up to Month 30	2.8% 1/36 • Up to Month 30
Injury, poisoning and procedural complications Ligament sprain	0.00% 0/99 • Up to Month 30	5.6% 10/177 • Up to Month 30	0.00% 0/36 • Up to Month 30
Respiratory, thoracic and mediastinal disorders Cough	10.1% 10/99 • Up to Month 30	4.0% 7/177 • Up to Month 30	0.00% 0/36 • Up to Month 30

Additional Information

Pfizer ClinicalTrials.gov Call Center

Pfizer Inc.

Phone: 1-800-718-1021

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Publication restrictions are in place

Restriction type: OTHER