Findings

What do systematic reviews say?

15 questions · 15 findings · 2 reviews

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No studies found 4 · 26.7%GRADE not reported 0 · 0%Very low 0 · 0%Low 1 · 6.7%Moderate 10 · 66.7%High 0 · 0%

Closest answers

The findings closest to your search, judged one by one by TypeSafe's Jev model.

Direct match · question page
- Intrathecal injected nusinersen vs sham procedure · Change in disability score
  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
Direct match · question page
- Intrathecal injected nusinersen vs sham procedure · Change in motor disability score ‐ response on HINE‐2 within one year after the onset of treatmente
  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
Direct match · question page
- Intrathecal injected nusinersen vs sham procedure · Time from beginning of treatment until death or full‐time ventilation
  Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
  no estimate extracted
  Studies not reported
  No studies found
Direct match · question page
- Intrathecal injected nusinersen vs sham procedure · Change in muscle strength
  Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
  no estimate extracted
  Studies not reported
  No studies found
Direct match · question page
- Intrathecal injected nusinersen vs sham procedure · Acquiring the ability to stand or walk
  Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
  RR 0.5 [0.03, 7.8]
  1 study · 126 participants
  Low

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

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

Intrathecal injected nusinersen vs sham

Muscle strength · 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

Intrathecal injected nusinersen vs sham

Acquiring the ability to stand or walk · children with SMA type II

Intrathecal injected nusinersen showed no clear difference in acquiring the ability to stand or walk compared with sham procedure (low certainty).

1 finding from 1 review

Drug treatment for spinal muscular atrophy types II and III 2020 · Cochrane Headline outcomes only
RR 0.5 [0.03, 7.8]
1 study · 126 participants
Low

Intrathecal injected nusinersen vs sham

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

Intrathecal injected nusinersen vs sham

Change in pulmonary function · 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

Intrathecal injected nusinersen vs sham

Time from beginning of treatment until death or full‐time ventilation · 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

Intrathecal injected nusinersen vs sham

Adverse events related to treatment · children with SMA type II

Intrathecal injected nusinersen showed no clear difference in adverse events related to treatment 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 0.9 [0.9, 1]
1 study · 126 participants
Moderate

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

Drug treatment for spinal muscular atrophy type I 2019 · Cochrane Headline outcomes only
HR 0.53 [0.32, 0.89]
1 study · 121 participants
Moderate

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

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

Drug treatment for spinal muscular atrophy type I 2019 · Cochrane Headline outcomes only
RR 0.7 [0.55, 0.89]
1 study · 121 participants
Moderate