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SMN2 Splicing Modifier
Long-Term Risdiplam for Spinal Muscular Atrophy
Recruiting3 awardsPhase 4
Washington, District of Columbia
This trial will study the long-term safety and effectiveness of the drug risdiplam in patients with spinal muscular atrophy. The trial will last for up to 5 years, and patients will be followed even if they discontinue the drug.
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