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 in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled 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: BN44621 https://forpatients.roche.com/ · 888-662-6728 (U.S. Only) · global-roche-genentech-trials@gene.com
Fastest response: use the inquiry form. No email attachments. https://www.gene.com/contact-us/submit-medical-inquiry
Always discuss trial participation with your own doctor first.
| University of Arkansas for Medical Sciences | Little Rock, Arkansas, United States | Recruiting |
| Valley Children's Hospital | Madera, California, United States | Recruiting |
| Stanford Univ Medical Center | Palo Alto, California, 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 |
| Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids, Michigan, United States | Recruiting |
| Children'S Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | Recruiting |
| University of Texas Southwestern Medical Center | Dallas, Texas, United States | Recruiting |
| Children's Hospital of the King's Daughter | Norfolk, Virginia, United States | Withdrawn |
| Charité - Universitätsmedizin Berlin SPZ Abteilung Neuropaediatrie | Berlin, Germany | Recruiting |
| UKGM Standort Gießen | Giessen, Germany | Recruiting |
+ 7 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05861999