Findings

What do systematic reviews say?

16 questions · 20 findings · 5 reviews · Neuromuscular

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No studies found 2 · 10%GRADE not reported 0 · 0%Very low 5 · 25%Low 7 · 35%Moderate 5 · 25%High 1 · 5%

Oral creatine vs placebo

Change in disability score · children with SMA types II and III

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 40 participants
Moderate

Oral hydroxyurea vs placebo

Change in disability score · children and adults with SMA types II and III

2 findings from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 57 participants
Low
Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 38 participants
Moderate

Oral olesoxime vs placebo

Change in disability score · non‐ambulatory children and adolescents with SMA types II and III

4 findings from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 160 participants
Very low
Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 160 participants
Very low
Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 1.43
1 study · 160 participants
Very low

Show 1 more finding

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 160 participants
Low

Oral phenylbutyrate vs placebo

Change in disability score · children with SMA type II

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 90 participants
Low

Subcutaneous somatotropin vs placebo

Change in disability score · children and adults with SMA types II and III

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
19 participants
Very low

Intravenous thyrotropin releasing hormone vs placebo

Change in disability score · children with SMA types II and III

The review found no eligible studies for this question.

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Oral valproic acid plus acetyl‐L‐carnitine vs placebo

Change in disability score · non‐ambulatory children with SMA types II and III

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 61 participants
Moderate

Oral valproic acid vs placebo

Change in disability score · ambulatory adults with SMA type III

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
31 participants
Low

Oral gabapentin vs placebo

Change in disability score · adults with SMA types II and III

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 66 participants
Low

Riluzole vs placebo

Change in motor score after two years · children with SMA type I

The review found no eligible studies for this question.

1 finding from 1 review

Drug treatment for spinal muscular atrophy type I 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

IVIg vs placebo

Change in mean disability score on Rankin scale · people with CIDP

1 finding from 1 review

Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy 2024 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 90 participants
High

IVIg vs placebo

Change in mean disability score at 24 weeks or later · people with CIDP

1 finding from 1 review

Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy 2024 · Cochrane Headline outcomes only
no estimate extracted
1 study · 117 participants
Moderate

Diflunisal vs placebo

Disability due to FAP progression (expressed as the change in Kumamoto Score) Scale from: 0 (normal) to 102 (worst disease severity). · people with TTR‐FAP

1 finding from 1 review

Pharmacological treatment for familial amyloid polyneuropathy 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 130 participants
Low

α‐tocopheryl quinone vs placebo

Longer‐term (minimum 1 month) change in generic quality of life scores · people with speech disorder resulting from hereditary ataxias

1 finding from 1 review

Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes 2014 · Cochrane Headline outcomes only
no estimate extracted
1 study · 19 participants
Moderate

Erythropoietin vs placebo

Longer‐term (minimum 1 month) change in generic quality of life scores · people with speech disorder resulting from hereditary ataxias

1 finding from 1 review

Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes 2014 · Cochrane Headline outcomes only
no estimate extracted
1 study · 16 participants
Low

Physiotherapy and occupational therapy vs placebo

Short‐term (1 week) change in quality of life scores related to communication as measured by validated communication assessments Functional Independence Measure (total). Scale from: 0 to 126. Higher scores indicate less disability · people with speech disorder resulting from hereditary ataxias

1 finding from 1 review

Treatment for speech disorder in Friedreich ataxia and other hereditary ataxia syndromes 2014 · Cochrane Headline outcomes only
no estimate extracted
1 study · 42 participants
Very low