Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
Status:
Not yet recruiting
Trial end date:
2029-02-01
Target enrollment:
Participant gender:
Summary
A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term
safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the
Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular
atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment
or until withdrawal of consent, loss to follow-up, or death.