Findings

What do systematic reviews say?

1,138 questions · 1,154 findings · 139 reviews · Headline outcomes only

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No studies found 184 · 15.9%GRADE not reported 183 · 15.9%Very low 273 · 23.7%Low 296 · 25.6%Moderate 171 · 14.8%High 47 · 4.1%

Oral gabapentin vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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 · 84 participants
Moderate

Oral gabapentin vs placebo

Adverse events related to treatment · 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 · 65 participants
Low

Oral hydroxyurea vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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
1 study · 57 participants
Moderate

Oral hydroxyurea vs placebo

Adverse events related to treatment · 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
1 study · 57 participants
Moderate

Intrathecal injected nusinersen vs sham

Time from beginning of treatment until death or full‐time ventilation · children with SMA type II

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

Intrathecal injected nusinersen vs sham

Adverse events related to treatment · children with SMA type II

Intrathecal injected nusinersen showed no clear difference in adverse events related to treatment compared with sham procedure (moderate certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 0.9 [0.9, 1]
1 study · 126 participants
Moderate

Oral olesoxime vs placebo

Time from beginning of treatment until death or full‐time ventilation · non‐ambulatory children and adolescents 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 · 160 participants
Moderate

Oral olesoxime vs placebo

Adverse events related to treatment · non‐ambulatory children and adolescents with SMA types II and III

Oral olesoxime probably leads to lower adverse events related to treatment compared with placebo (moderate certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 0.95 [0.91, 0.99]
1 study · 165 participants
Moderate

Oral phenylbutyrate vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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 · 107 participants
Low

Oral phenylbutyrate vs placebo

Adverse events related to treatment · children with SMA type II

Oral phenylbutyrate probably leads to higher adverse events related to treatment compared with placebo (moderate certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 3.1 [1.25, 7.84]
1 study · 107 participants
Moderate

Subcutaneous somatotropin vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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
Low

Subcutaneous somatotropin vs placebo

Adverse events related to treatment · children and adults with SMA types II and III

Subcutaneous somatotropin showed no clear difference in adverse events related to treatment compared with placebo (low certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 1.57 [0.78, 3.17]
19 participants
Low

Oral creatine vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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

Intravenous thyrotropin releasing hormone vs placebo

Adverse events related to treatment · 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 · 9 participants
Very low

Oral valproic acid plus acetyl‐L‐carnitine vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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 plus acetyl‐L‐carnitine vs placebo

Adverse events related to treatment · non‐ambulatory children with SMA types II and III

Oral valproic acid plus acetyl‐L‐carnitine showed no clear difference in adverse events related to treatment compared with placebo (moderate certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 1.32 [0.92, 1.89]
1 study · 61 participants
Moderate

Oral creatine vs placebo

Adverse events related to treatment · 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
Low

Oral valproic acid vs placebo

Time from beginning of treatment until death or full‐time ventilation · 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
33 participants
Low

Oral valproic acid vs placebo

Adverse events related to treatment · ambulatory adults with SMA type III

Oral valproic acid showed no clear difference in adverse events related to treatment compared with placebo (low certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 0.8 [0.44, 1.44]
33 participants
Low

Riluzole vs placebo

Adverse events attributable to treatment · children with SMA type I

1 finding from 1 review

Drug treatment for spinal muscular atrophy type I 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 10 participants
Very low