A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Conditions
Muscular Atrophy, Spinal
Simple summaryAI-assisted
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Locations
University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
Children's Hospital of Colorado, Aurora, Colorado, United States
University of Florida Pediatrics, Gainesville, Florida, United States
Children's Healthcare of Atlanta Center for Advanced Pediatrics, Atlanta, Georgia, United States
Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
Helen DeVos Children's Hospital at Spectrum Health, Grand Rapids, Michigan, United States
Columbia University Medical Center, New York, New York, United States
Children'S Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
The University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, United States
Cook Children's Jane and John Justin Neurosciences Center, Fort Worth, Texas, United States
Children's Hospital of the King's Daughter, Norfolk, Virginia, United States
Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie, Berlin, Germany
UKGM Standort Gießen, Giessen, Germany
Uniwersyteckie Centrum Kliniczne, Uniwersyteckie Centrum Kliniczne, Poland
Instytut Pomnik Centrum Zdrowia Dziecka, Warsaw, Poland
Great Ormond Street Hospital For Children, London, United Kingdom
Study leads
Clinical Trials
Study Director
Hoffmann-La Roche