This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have…
- Investigators
- Carmen Leon Astudillo, Renata Shih
- Ages
- 3 Months - 24 Months
- Sexes
- All