Findings

What do systematic reviews say?

13 questions · 17 findings · 2 reviews

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No studies found 2 · 11.8%GRADE not reported 0 · 0%Very low 4 · 23.5%Low 5 · 29.4%Moderate 6 · 35.3%High 0 · 0%

Intrathecal injected nusinersen vs sham

Change in motor score after one year · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen probably leads to higher change in motor disability score ‐ response on HINE‐2 within one… compared with sham procedure (moderate certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy type I 2019 · Cochrane Headline outcomes only
RR 38.51 [2.43, 610.14]
1 study · 110 participants
Moderate

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

Intrathecal injected nusinersen vs sham

Change in disability score · children with SMA type II

Intrathecal injected nusinersen probably leads to higher change in disability score 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
MD 5.9 [3.7, 8.1]
1 study · 126 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

Intrathecal injected nusinersen vs sham

Change in disability score (3 point‐change) · children with SMA type II

Intrathecal injected nusinersen showed no clear difference in change in disability score (3 point‐change) 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 1.8 [0.99, 3.1]
1 study · 126 participants
Moderate