Findings

What do systematic reviews say?

461 questions · 472 findings · 42 reviews · Neuromuscular

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No studies found 93 · 19.7%GRADE not reported 81 · 17.2%Very low 99 · 21%Low 103 · 21.8%Moderate 75 · 15.9%High 21 · 4.4%

Oral phenylbutyrate vs placebo

Acquiring the ability to stand or walk · 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

Oral phenylbutyrate vs placebo

Change in quality of life · 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

Oral phenylbutyrate vs placebo

Change in pulmonary function · 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 · 67 participants
Low

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

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

Subcutaneous somatotropin vs placebo

Muscle strength · 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

Oral creatine vs placebo

Change in pulmonary function · 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 · 23 participants
Low

Subcutaneous somatotropin vs placebo

Acquiring the ability to stand or walk · children and adults 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

Subcutaneous somatotropin vs placebo

Change in quality of life · 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

Subcutaneous somatotropin vs placebo

Change in pulmonary function · 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

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

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

Intravenous thyrotropin releasing hormone vs placebo

Muscle strength · 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

Intravenous thyrotropin releasing hormone vs placebo

Acquiring the ability to stand or walk · 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

Intravenous thyrotropin releasing hormone vs placebo

Change in quality of life · 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

Intravenous thyrotropin releasing hormone vs placebo

Change in pulmonary function · 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 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

Time from birth 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 · 9 participants
Low