Russman 2003.

Methods	Randomised (2:1 ratio), placebo‐controlled trial The investigational review boards of the participating centres approved the protocol and consent forms
Participants	10 children (mean age at inclusion 10 months; range 3 to 15 months) who fulfilled international classification criteria for SMA type I and have a homozygous deletion of the SMN1 gene
Interventions	Riluzole orally, around 107 mg/m²/day or placebo  Duration of treatment 9 months, follow‐up 12 months Power was calculated on 30 included participants (2:1 ratio)
Outcomes	Number of children who died Age at death Adverse events
Funding	The study was funded in part by Rhone‐Poulenc‐Rorer, the manufacturer of riluzole, who provided the medication. Support was withdrawn when the company was taken over by Aventis.
Conflicts of interest	Not stated
Notes	Enrolment goal was 30 children with SMA type I, but only 10 children were included until funding was withdrawn
Risk of bias
Bias	Authors' judgement	Support for judgement
Random sequence generation (selection bias)	High risk	Randomly assigned (2:1), no method reported. Infants receiving riluzole were older at diagnosis (5.2 months) than those who received placebo (1.2 months) and older at enrolment (9.3 versus 4.3 months)
Allocation concealment (selection bias)	Unclear risk	Not reported
Blinding of participants and personnel (performance bias) All outcomes	Unclear risk	Unknown
Blinding of outcome assessment (detection bias) All outcomes	Unclear risk	Unknown
Incomplete outcome data (attrition bias) All outcomes	Low risk	All 10 participants recruited provided outcome data
Selective reporting (reporting bias)	Low risk	There is only one possible way in which the outcome domains can be measured (hence there is no opportunity to select from multiple measures).
Other bias	Low risk	Trial was terminated prematurely

CHOP INTEND: Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders; CSF: cerebrospinal fluid; ECG: electrocardiogram: HINE: Hammersmith Infant Neurological Examination; SMA: spinal muscular atrophy; SMN: survival motor neuron