Condition: Muscular Atrophy, Spinal · Sponsor: Hoffmann-La Roche
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.
This description comes directly from the study's public registry record.
Reference Study ID Number: BN44620 https://forpatients.roche.com/ · 888-662-6728 (U.S. Only) · global-roche-genentech-trials@gene.com
Always discuss trial participation with your own doctor first.
| University of Arkansas for Medical Sciences | Little Rock, Arkansas, United States | Recruiting |
| Children's Hospital of Colorado | Aurora, Colorado, United States | Recruiting |
| University of Florida Pediatrics | Gainesville, Florida, United States | Recruiting |
| Children's Healthcare of Atlanta Center for Advanced Pediatrics | Atlanta, Georgia, United States | Recruiting |
| Ann and Robert H. Lurie Children Hospital of Chicago | Chicago, Illinois, United States | Not Yet Recruiting |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids, Michigan, United States | Recruiting |
| Columbia University Medical Center | New York, New York, United States | Recruiting |
| Children'S Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | Recruiting |
| The University of Texas Southwestern Medical Center at Dallas | Dallas, Texas, United States | Recruiting |
| Cook Children's Jane and John Justin Neurosciences Center | Fort Worth, Texas, United States | Recruiting |
| Children's Hospital of the King's Daughter | Norfolk, Virginia, United States | Recruiting |
| Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie | Berlin, Germany | Recruiting |
+ 4 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05861986