Study Summary
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
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RisdiplamDRUG
Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| 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 |