- 26 infants participated in a risdiplam clinical trial.
- Risdiplam is an SMN2 pre-mRNA splicing modifier.
- Infants were aged 1-42 days at first dose.
- Primary endpoint included motor milestone achievement.
- 21 infants could sit unsupported for 30 seconds at 12 months.
- Early treatment supports survival without permanent ventilation.
- Treatment-related adverse events were mostly mild.
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