Search review questions

Measures of lung capacity over the short term (less than 3 months) · children and young males with DMD

Measures of lung capacity over the medium term (greater than 3 months but less than 1 year) Measured by: post‐intervention % predicted VC · children and young males with DMD

Measures of lung capacity over the long term (greater than 1 year) · children and young males with DMD

Physical function in carrying out activities of daily living · children and young males with DMD

Quality of life · children and young males with DMD

Measures of lung capacity over the medium term (greater than 3 months but less than 1 year) (change from baseline in % predicted FVC) · people with ALS

Number of unscheduled hospitalizations for episodes of chest infection or acute exacerbation of chronic respiratory failure within 1 year of randomization · children and young males with DMD

All adverse events · children and young males with DMD

Measures of lung capacity over the short term (less than 3 months) Measured as change from baseline in FVC (L) · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

Measures of lung capacity over the medium term (greater than 3 months but less than 1 year) · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

Measures of lung capacity over the long term (greater than 1 year) · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

Physical function in carrying out activities of daily living · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

Quality of life · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

Number of unscheduled hospitalisations for episodes of chest infection or acute exacerbation of chronic respiratory failure within 1 year of randomization · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

All adverse events · participants with limb‐girdle muscular dystrophy or Becker muscular dystrophy

Measures of lung capacity over the short term (less than 3 months) Measured by: post‐intervention TLC · people with myasthenia gravis

Measures of lung capacity over the long term (greater than 1 year) · people with ALS

Measures of lung capacity over the medium term (greater than 3 months but less than 1 year) · people with myasthenia gravis

Measures of lung capacity over the long term (greater than 1 year) · people with myasthenia gravis

Physical function in carrying out activities of daily living · people with myasthenia gravis

Quality of life · people with myasthenia gravis

Number of unscheduled hospitalizations for episodes of chest infection or acute exacerbation of chronic respiratory failure within 1 year of randomization · people with myasthenia gravis

All adverse events · people with myasthenia gravis

Physical function in carrying out activities of daily living over the medium term (3 to 12 months; change from baseline) · people with ALS

Quality of life (change from baseline) in the medium term · people with ALS

Number of unscheduled hospitalisations for episodes of chest infection or acute exacerbation of chronic respiratory failure within 1 year of randomization · people with ALS

All adverse events · people with ALS

Measures of lung capacity over the short term (less than 3 months) Measured by: post‐intervention TLC · children and young males with DMD

Measures of lung capacity over the medium term (greater than 3 months but less than 1 year) · children and young males with DMD

Functional impairment, assessed using a functional rating scale (change from baseline to 6 months): mean change in ALSFRS‐R score · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Functional impairment, assessed using a functional rating scale (change from baseline to 12 months) · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Muscle strength: manual muscle testing (mean change from baseline to 12 months) · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Respiratory function: change in FVC% (mean change from baseline to 12 months) · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Overall survival · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Adverse events, total · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Serious adverse events · people with probable or definite amyotrophic lateral sclerosis/motor neuron disease (treated with riluzole)

Walking distance on the 6MWT (m) (a higher score indicates better function) Change from baseline · children and adults with SMA type 3

Cardiopulmonary exercise capacity · children and adults with SMA type 3

Functional performance · children and adults with SMA type 3

Muscle strength (MMT total score) · children and adults with SMA type 3

Fatigue In children, · children and adults with SMA type 3

Physical activity ‐ change from baseline · children and adults with SMA type 3

Quality of life In children, · children and adults with SMA type 3

Serious adverse events leading to withdrawal · children and adults with SMA type 3

Improvement (greater than 50% of time spent) heel‐toe walking Measured by parents' perception of toe walking frequency more than 50% of the time. · children with ITW gait

Active range of motion of the ankle joint · children with ITW gait

Change from baseline in passive ankle dorsiflexion range of movement · children with ITW gait

Pain · children with ITW gait

Recurrence of ITW post‐treatment Assessed by improvement in toe walking based on Gait Analysis Classification An increase indicates improvement (an improvement indicated by a change in downgrading of the severe or 3, moderate or 2, and mild or 1 c... · children with ITW gait

Adverse events · children with ITW gait