A NATURAL HISTORY STUDY IN CHINESE MALE PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY

Age of Participants When They Failed to Walk

时间窗: Up to Month 30

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.

Age of Participants When They Failed to Stand

时间窗: 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.

Age of Participants When They Failed to Self-feed

时间窗: 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.

Change From Baseline in Northstar Ambulatory Assessment (NSAA) Total Score at Month 6: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in NSAA Total Score at Month 12: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in NSAA Total Score at Month 18: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in NSAA Total Score at Month 24: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in NSAA Total Score at Month 30: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Performance of Upper Limb (PUL) 2.0 Total Score at Month 6: Participants Aged >=10 Years

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in PUL 2.0 Total Score at Month 12: Participants Aged >=10 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in PUL 2.0 Total Score at Month 18: Participants Aged >=10 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in PUL 2.0 Total Score at Month 24: Participants Aged >=10 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in PUL 2.0 Total Score at Month 30: Participants Aged >=10 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Rise From Floor Velocity at Month 6: Ambulatory Participants Aged >=3 Years Only

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in Rise From Floor Velocity at Month 12: Ambulatory Participants Aged >=3 Years Only

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in Rise From Floor Velocity at Month 18: Ambulatory Participants Aged >=3 Years Only

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in Rise From Floor Velocity at Month 24: Ambulatory Participants Aged >=3 Years Only

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in Rise From Floor Velocity at Month 30: Ambulatory Participants Aged >=3 Years Only

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in 10 Meter Walk or Run Velocity at Month 6: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in 10 Meter Walk or Run Velocity at Month 12: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in 10 Meter Walk or Run Velocity at Month 18: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in 10 Meter Walk or Run Velocity at Month 24: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in 10 Meter Walk or Run Velocity at Month 30: Ambulatory Participants Aged >=3 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Knee Extension of Muscle Strength at Month 6: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in Knee Extension of Muscle Strength at Month 12: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in Knee Extension of Muscle Strength at Month 18: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in Knee Extension of Muscle Strength at Month 24: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in Knee Extension of Muscle Strength at Month 30: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Elbow Flexion of Muscle Strength at Month 6: Participants Aged >=5 Years

时间窗: 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.

Change From Baseline in Elbow Flexion of Muscle Strength at Month 12: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in Elbow Flexion of Muscle Strength at Month 18: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in Elbow Flexion of Muscle Strength at Month 24: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in Elbow Flexion of Muscle Strength at Month 30: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Elbow Extension of Muscle Strength at Month 6: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in Elbow Extension Muscle Strength at Month 24: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in Elbow Extension Muscle Strength at Month 12: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in Elbow Extension Muscle Strength at Month 18: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in Shoulder Abduction of Muscle Strength at Month 30: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Elbow Extension Muscle Strength at Month 30: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Shoulder Abduction of Muscle Strength at Month 6: Participants Aged >=5 Years

时间窗: 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.

Change From Baseline in Shoulder Abduction of Muscle Strength at Month 12: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in Shoulder Abduction of Muscle Strength at Month 18: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in Shoulder Abduction of Muscle Strength at Month 24: Participants Aged >=5 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in Range of Motion (ROM) at Bilateral Ankles at Month 6

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in ROM at Bilateral Ankles at Month 12

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in ROM at Bilateral Ankles at Month 18

时间窗: Baseline (Day 1) and Month 18

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.

Change From Baseline in ROM at Bilateral Ankles at Month 24

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in ROM at Bilateral Ankles at Month 30

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in ROM at Bilateral Elbows at Month 6

时间窗: Baseline (Day 1) and Month 6

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.

Change From Baseline in ROM at Bilateral Elbows at Month 12

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in ROM at Bilateral Elbows at Month 18

时间窗: 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.

Change From Baseline in ROM at Bilateral Elbows at Month 24

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in ROM at Bilateral Elbows at Month 30

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in Percent Predicted Forced Vital Capacity (%pFVC) at Month 12: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in %pFVC at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 12

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in %pFVC at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 24

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in %pFVC at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 30

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%pFEV1) at Month 12: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in %pFEV1 at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 12

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in %pFEV1 at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 24

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in %pFEV1 at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 30

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in Maximum Inspiratory Pressure at Month 12: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 12

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

Change From Baseline in Maximum Inspiratory Pressure at Month 24: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 24

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

Change From Baseline in Maximum Inspiratory Pressure at Month 30: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 30

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

Change From Baseline in Maximum Expiratory Pressure at Month 12: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 12

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

Change From Baseline in Maximum Expiratory Pressure at Month 24: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 24

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

Change From Baseline in Maximum Expiratory Pressure at Month 30: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 30

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

Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 12

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

Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 24

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

Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 30

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

Change From Baseline in Peak Cough Flow at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 12

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

Change From Baseline in Peak Cough Flow at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 24

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

Change From Baseline in Peak Cough Flow at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 30

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

Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Month 12: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 12

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.

Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 24

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.

Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years

时间窗: Baseline (Day 1) and Month 30

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.

Change From Baseline in LVEF at Month 12: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 12

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in LVEF at Month 24: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 24

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in LVEF at Month 30: Participants Aged >=6 Years (Unplanned Analysis)

时间窗: Baseline (Day 1) and Month 30

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. Incorrect data were addressed by unplanned analysis.

Change From Baseline in Wechsler Intelligence Scale for Children (WISC)-IV Score at Month 24: Ambulatory Participants >= 6 to <=16 Years

时间窗: Baseline (Day 1) and Month 24

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.