Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

1,139 questions · 1,173 findings · 89 reviews · Neuromuscular

No studies found 302 · 25.7%GRADE not reported 119 · 10.1%Very low 267 · 22.8%Low 239 · 20.4%Moderate 177 · 15.1%High 69 · 5.9%

Intrathecal injected nusinersen vs sham

Time from birth until death or full‐time ventilation · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen probably leads to lower time from birth until death or full‐time ventilationa compared with sham procedure (moderate certainty).

1 finding from 1 review

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

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

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

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

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

Intrathecal injected nusinersen vs sham

Severe adverse events attributable to treatment Measured as severe adverse events (all) · infants with SMA and 2 SMN2 copies

Intrathecal injected nusinersen probably leads to lower severe adverse events attributable to treatment Measured as severe… compared with sham procedure (moderate certainty).

1 finding from 1 review

Intervention not reported

In‐hospital death from any cause occurring within 4 weeks of randomization or the beginning of treatment · individuals with infant botulism
1 finding from 1 review