Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

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

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%

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

Proportion of patients who discontinued treatment due to adverse events (28 weeks trial period) · Patients between five and 15 years old with a diagnosis of BECTS

The intervention showed no clear difference in proportion of patients who discontinued treatment due to adverse… (low certainty).

1 finding from 1 review

Intervention not reported

Proportion of patients who discontinued treatment due to adverse events (96 weeks trial period) · Patients with a clinical and EEG diagnosis of BECTS

It is very uncertain whether the intervention changes proportion of patients who discontinued treatment due to adverse… (very low certainty).

1 finding from 1 review

Intervention not reported

Proportion of patients who discontinued treatment due to adverse events ( · Patients between three and 12 years old with clinical and EEG diagnosis of BECTS

The intervention showed no clear difference in proportion of patients who discontinued treatment due to adverse… (low certainty).

1 finding from 1 review