Search review questions

Adverse effects (during study) · people with speech disorder resulting from hereditary ataxia

Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) · people with speech disorder resulting from hereditary ataxia

Economic outcomes · people with speech disorder resulting from hereditary ataxia

Short‐term (1 week) percentage change (improvement) in overall speech production · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production ICARS. · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Longer‐term (minimum 1 month) change in generic quality of life scores · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Longer‐term burdens (minimum 1 month) (for example demands on caregivers, frequency of tests and restrictions on lifestyle) · people with speech disorder resulting from hereditary ataxias

Adverse effects (during study) · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Economic outcomes · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Short‐term (1 week) percentage change (improvement) in overall speech production · people with speech disorder resulting from hereditary ataxia

Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production · people with speech disorder resulting from hereditary ataxia

Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments · people with speech disorder resulting from hereditary ataxia

Longer‐term (minimum 1 month) change in generic quality of life scores SF‐36 scale from: 0 to 100. Higher score indicate less disability. · people with speech disorder resulting from hereditary ataxia

Adverse effects (during study) · people with speech disorder resulting from hereditary ataxia

Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) · people with speech disorder resulting from hereditary ataxia

Economic outcomes · people with speech disorder resulting from hereditary ataxia

Economic outcomes · people with speech disorder resulting from hereditary ataxias

Short‐term (1 week) percentage change (improvement) in overall speech production · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Short‐term (within 1 month) change in isolated movement, objective and subjective measures of speech production SARA. Scale from: 0 to 48. · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Short‐term (within 1 month) change in quality of life scores related to communication as measured by validated communication assessments · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Longer‐term (minimum 1 month) change in generic quality of life scores SF‐36. Scale from 0 to 100. Higher scores indicate less disability. · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Adverse effects (during study) · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Longer‐term burdens (minimum 1 month) (for example, demands on caregivers, frequency of tests, restrictions on lifestyle) · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Economic outcomes · people with speech disorder in Friedreich ataxia and other hereditary ataxias

Short‐term (1 week) percentage change (improvement) in overall speech production · people with speech disorder resulting from hereditary ataxia

Short‐term (1 week) change in isolated movement, objective and subjective measures of speech production Inherited Ataxias Clinical Rating Scale (IACRS) · people with speech disorder resulting from hereditary ataxia

Immediate improvement in pain relief Number of participants improved (good/very good efficacy) on a self assessed categorical 'overall efficacy score' (0 = no efficacy to 4 = very good) · patients with trigeminal neuralgia

50% pain reduction 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Quality of life assessment 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Adverse effects (not reported) · patients with trigeminal neuralgia

Pain relief (TN score) · patients with trigeminal neuralgia

50% pain reduction 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Quality of life assessment 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Adverse effects The number of participants with adverse effects · patients with trigeminal neuralgia

50% pain reduction 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Quality of life assessment 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Adverse effects (not reported) · patients with trigeminal neuralgia

Pain relief (frequency and intensity) from the baseline 0 to 10‐point scale · patients with trigeminal neuralgia

50% pain reduction 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Quality of life assessment 12 weeks after the start of treatment Not measured · patients with trigeminal neuralgia

Adverse effects · patients with trigeminal neuralgia

Immediate improvement in pain relief Number of participants improved (≥ 2 level decrease in pain on 10‐point verbal rating scale, or pain gone or rare) · patients with trigeminal neuralgia

Maximum voluntary contraction in dystrophinopathies % · patients with muscular dystrophies

Adverse events in glycogen storage disease type V · patients with metabolic myopathies

Aggregate functional performance time (AFPT), intention‐to‐treat analysis % · patients with idiopathic inflammatory myopathies

AFPT, valid compliant completer analysis % · patients with idiopathic inflammatory myopathies

Adverse events · patients with idiopathic inflammatory myopathies

Maximum voluntary contraction in myotonic dystrophy type 1 % · patients with muscular dystrophies