Summary of findings 2. Riluzole compared to placebo for children with SMA type I.

Riluzole compared to placebo for children with SMA type I
Patient or population: children with SMA type I Setting: outpatient clinic Intervention: riluzole Comparison: placebo
Outcomes	Impact	№ of participants (studies)	Certainty of the evidence (GRADE)	Commments
Time from birth until death or full‐time ventilation Follow‐up: range 1 month to 64 months	In the 3 children in the placebo group, the median age at death was 8 months (range 6 to 13 months). 4/7 children treated with riluzole died during the trial, at a median age of 17 months (range 5 to 25 months); the remaining 3 children treated with riluzole were still alive at 30, 48 and 64 months.	10 (1 RCT)	⊕⊝⊝⊝ Very lowa
Acquisition of head control	‐	‐	‐	Not measured
Acquisition of the ability to sit Follow‐up: range 1 month to 64 months	None of the children in the riluzole or the placebo group acquired the ability to sit (follow‐up was extended up to 30 to 64 months in the children treated with riluzole).	10 (1 RCT)	⊕⊝⊝⊝ Very lowa
Acquisition of the ability to stand	‐	‐	‐	Not measured
Change in motor disability score	‐	‐	‐	Not measured
Adverse events attributable to treatment Follow‐up: range 1 month to 9 months	No adverse events occurred in children treated with placebo or riluzole.	10 (1 RCT)	⊕⊝⊝⊝ Very lowa
Severe adverse events attributable to treatment Follow‐up: range 1 months to 9 months	No adverse events occurred in children treated with placebo or riluzole.	10 (1 RCT)	⊕⊝⊝⊝ Very lowa
*The risk in the intervention group (and its 95% CI) is based on the assumed risk in the comparison group and the relative effect of the intervention (and its 95% CI). RCT: randomised controlled trial; SMA: spinal muscular atrophy
GRADE Working Group grades of evidence High certainty: we are very confident that the true effect lies close to that of the estimate of the effect. Moderate certainty: we are moderately confident in the effect estimate; the true effect is likely to be close to the estimate of the effect, but there is a possibility that it is substantially different. Low certainty: our confidence in the effect estimate is limited; the true effect may be substantially different from the estimate of the effect. Very low certainty: we have very little confidence in the effect estimate; the true effect is likely to be substantially different from the estimate of effect.

^aWe downgraded the certainty of the evidence three levels: twice for serious imprecision, because of the small cohort, and once for risk of bias as there were baseline differences between treatment and control groups.