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. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.

Conditions

  • Spinal Muscular Atrophy

Intervention

  • Risdiplam

Principal Investigator

  • Michelson, David J

Sponsor

  • Genentech, Inc.

*The source of data for this particular study is "ClinicalTrials.gov" and was last updated on 05/16/2024. Loma Linda University does not make any warranties, expressed or implied, with respect to such data, and, furthermore, assumes no liability for any party's use, or the results of such use of the data.

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