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Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Monitoring Risdiplam effects in Spinal Muscular Atrophy patients long-term.

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Phase 4
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This study looks at the long-term safety and how well the medicine risdiplam works for people with spinal muscular atrophy (SMA). SMA is a disease that affects the muscles, making them weak and small. Risdiplam is a treatment option for SMA. The study will follow participants for up to five years, or until they decide to stop, are lost to follow-up, or pass away. Even if they stop taking risdiplam, they can still be part of the study if they agree to continue with the check-ups.

Participants need to have a confirmed diagnosis of a certain type of SMA (5q-autosomal recessive). They should be taking risdiplam based on their doctor's advice, following the U.S. guidelines. People who are allergic to risdiplam or have been in previous major risdiplam trials cannot join.

  • The study lasts up to 5 years.
  • Participants can stay in the study even if they stop taking the medicine.
  • Participants must not be allergic to risdiplam.
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Study details
    Spinal Muscular Atrophy

NCT05232929

Genentech, Inc.

9 February 2025

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