Risdiplam Study for Pediatric Spinal Muscular Atrophy (SMA)

This study checks how well the drug risdiplam works and if it's safe for young children with Spinal Muscular Atrophy (SMA), a condition that affects muscles and movement. It is for kids under 2 years old who have been diagnosed with SMA and have 2 copies of the SMN2 gene, which is linked to this condition. These kids must have already received a gene therapy called onasemnogene abeparvovec but are not getting better or are getting worse.

Key Points:

• The study involves only one group of patients and takes place at multiple centers.
• Participants need to be under 2 years old and have shown a plateau or decline in physical function after gene therapy.
• Some exclusions: recent hospital stays, other investigational therapies, or severe medical conditions.

This study is important for understanding how risdiplam can help children with SMA. If your child meets the criteria, joining this study could provide access to new treatments and contribute to scientific research.