Abstract
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 dose.
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 dose.
Stanley Manne Children's Research Institute
Louis A. Simpson and Kimberly K. Querrey Biomedical Research Center
303 East Superior Street
Chicago, IL 60611
