Findings

What do systematic reviews say?

1,181 questions · 1,197 findings · 144 reviews

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No studies found 196 · 16.4%GRADE not reported 213 · 17.8%Very low 274 · 22.9%Low 296 · 24.7%Moderate 171 · 14.3%High 47 · 3.9%

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

Riluzole vs placebo

Severe 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

Intrathecal injected nusinersen vs sham

Acquisition of head control · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen probably leads to higher acquisition of head control within one year after the onset of… 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 16.95 [1.04, 274.84]
1 study · 110 participants
Moderate

Intrathecal injected nusinersen vs sham

Acquisition of the ability to sit · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen showed no clear difference in acquisition of the ability to sit within one year after the onset of… 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 6.68 [0.39, 115.38]
1 study · 110 participants
Moderate

Intrathecal injected nusinersen vs sham

Acquisition of the ability to stand · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen showed no clear difference in acquisition of the ability to stand within one year after the onset… 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 1.54 [0.06, 36.92]
1 study · 110 participants
Moderate

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

Intrathecal injected nusinersen vs sham

Adverse events attributable to treatment · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen showed no clear difference in adverse events attributable to treatment Measured as adverse events… 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 0.99 [0.92, 1.05]
1 study · 121 participants
Moderate