New study tracks Risdiplam's Long-Term effects in SMA patients
NCT ID NCT05232929
First seen Dec 08, 2025 · Last updated May 08, 2026 · Updated 18 times
Summary
This study follows about 400 adults and children with spinal muscular atrophy (SMA) who are taking risdiplam (Evrysdi) to see how safe and effective it is over the long term. Participants will be monitored for about 2 years, and the study tracks side effects and changes in their condition. Risdiplam is an ongoing treatment, not a cure, so patients take it indefinitely to manage their disease.
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Advent Health Orlando
Winter Park, Florida, 32789, United States
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All Children's Research Institute, Inc.
St. Petersburg, Florida, 33701, United States
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Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
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Central Texas Neurology Consultants
Round Rock, Texas, 78681, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90010, United States
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, 53201, United States
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Cincinnati Childrens Hospital
Cincinnati, Ohio, 45229, United States
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Columbia University Med Center
New York, New York, 10032, United States
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Gillette Children's Specialty Healthcare
Minnetonka, Minnesota, 55343, United States
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Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, 49503, United States
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Instituto de Rehabilitación del Caribe
Santurce, 00912, Puerto Rico
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Loma Linda University Health Care
Loma Linda, California, 92354, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Medstar Georgetown University Hospital
Washington D.C., District of Columbia, 20007, United States
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Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229, United States
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MultiCare Health System Institute for Research and Innovation
Tacoma, Washington, 98405, United States
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NYU Hospital for Joint Diseases
New York, New York, 10003, United States
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Nemour's Children's Hospital, Florida
Orlando, Florida, 32827, United States
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Neurology & Neuromuscular Care Center
Denton, Texas, 76208, United States
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Norton Children's Hospital
Louisville, Kentucky, 40202, United States
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Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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UBC (Remote Coordinating Center, no physical facility)
Morgantown, West Virginia, 26508, United States
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University California - Irvine
Orange, California, 92868, United States
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University Of Utah
Salt Lake City, Utah, 84108, United States
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University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72103, United States
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University of California Davis Medical Center
Sacramento, California, 95817, United States
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University of Colorado
Aurora, Colorado, 80045, United States
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University of Kentucky Medical Center
Lexington, Kentucky, 40536, United States
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University of Michigan Pediatric Rehabilitation Center
Ann Arbor, Michigan, 48109, United States
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University of Texas at Austin Health sciences, Dell Medical School
Austin, Texas, 78723, United States
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University of Virginia Children?s Hospital
Charlottesville, Virginia, 22903, United States
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Valley Children's Hospital
Madera, California, 93636, United States
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Washington University;Wash Uni. Sch. Of Med
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.