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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Evaluating Risdiplam's effects in kids with Spinal Muscular Atrophy post-gene therapy.

Recruiting
3-24 years
All
Phase 4

Spinal Muscular Atrophy (SMA) is a genetic condition that affects muscles, making them weak. In this study, doctors are testing a medicine called risdiplam as an early treatment for young children under 2 years old who have SMA and have 2 copies of a specific gene called SMN2. These children have already received a gene therapy called onasemnogene abeparvovec which is given to help their muscles before or after symptoms start showing.

The study is open-label, meaning everyone knows what treatment is given, and single-arm, meaning all participants get the same treatment. To join, children must have had the gene therapy 3-7 months before starting this study. They should not have any serious new symptoms after the gene therapy.

  • The study is for children under 2 years old.
  • They must have already had gene therapy (onasemnogene abeparvovec).
  • Participants should not have serious breathing or feeding issues.
Study details
    Muscular Atrophy
    Spinal

NCT05861986

Hoffmann-La Roche

21 June 2025

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