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A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dos…

Muscular Atrophy, Spinal

Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05808764