SMA drug risdiplam under Long-Term watch: will it hold up?
NCT ID NCT05232929
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 403 adults and children with spinal muscular atrophy who are already taking risdiplam (Evrysdi) as prescribed by their doctor. Researchers will track side effects and how well the drug works over about two years. The goal is to see if risdiplam remains safe and effective for long-term use.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
risdiplam (Evrysdi)
What this could lead to
If successful, this study will confirm that risdiplam remains safe and effective for long-term use in managing spinal muscular atrophy.
What could go wrong
This is an observational follow-up, not a test of a new treatment. It may not prove long-term benefit beyond what is already known.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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