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Spinal Muscular Atrophy clinical trials at UC Irvine

1 research study open to eligible people

Showing trials for
  • Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

    open to all eligible people

    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.

    Orange, California and other locations

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