Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
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 Information (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for approximately 24 months from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study if they agree to continue participating in the follow-up assessments.
An optional sub-study was planned to assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. This substudy was withdrawn upon implementation of protocol version 4.
Inclusion Criteria:
Exclusion Criteria:
Austin, Texas 78723, United States
Tacoma, Washington 98405, United States
Morgantown, West Virginia 26508, United States