This study involves people with a condition called **Spinal Muscular Atrophy (SMA)**, a disease that weakens muscles. The study will look at how safe and effective a medicine called **risdiplam** is over a long time. Risdiplam is a medicine approved by the U.S. FDA (Food and Drug Administration) to help with SMA. The study follows participants for a long time, until they choose to leave, are lost to follow-up, or pass away. Even if participants stop taking risdiplam, they may continue in the study for follow-up checks. There’s also an optional sub-study using smartphones and sensors to check movements and swallowing.
To join, you must have a clinical diagnosis of SMA and be on risdiplam based on doctor's judgment. For the sub-study, participants must be at least 10 years old, comfortable using smartphones, and able to complete specific tests. Participants who are allergic to risdiplam or have certain mental health issues cannot join.
- The study will last as long as participants are willing and able.
- Participants may need to visit the clinic for assessments.
- There might be some risks, especially for those with specific health conditions.