Study Purpose: This study looks at how well a medicine called risdiplam works and how safe it is for young children with Spinal Muscular Atrophy (SMA). SMA is a genetic problem that makes muscles weak. The study focuses on kids under 2 years old who have already tried another treatment called onasemnogene abeparvovec but stopped improving or got worse.
Eligibility: To join, kids must be under 2 years old and have a confirmed diagnosis of SMA with 2 copies of a gene called SMN2. They must have had onasemnogene abeparvovec and shown decreased ability to do things like swallow or move.
- Length: The study requires visits over several months to check progress.
- Treatment: Participants will receive risdiplam, which is not tested on kids who had other similar treatments.
- Risks: Eligibility excludes kids with certain health issues, like needing a feeding tube or recent hospital stays.
This study is conducted across multiple centers and does not have a placebo group, so all participants will receive the treatment. This is an open-label study, meaning both doctors and families know the treatment being given.