Real-World DMD drug effects under the microscope
NCT ID NCT06606340
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 30 times
Summary
This study follows 300 people with Duchenne muscular dystrophy who are already taking eteplirsen, golodirsen, or casimersen in everyday medical care. Researchers will collect information on movement, breathing, and heart function over time to see how these treatments work in the real world. The goal is to better understand long-term outcomes, not to test a new drug.
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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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Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202, United States
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Atrium Health Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Columbia University Medical Center - PIN
New York, New York, 10032, United States
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Cook Children's Hospital
Fort Worth, Texas, 76104, United States
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Duke Lenox Baker Children's Hospital
Durham, North Carolina, 27705, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21287, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
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UVA Children's Hospital
Charlottesville, Virginia, 22908, United States
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University Of Iowa Hospitals And Clinics
Iowa City, Iowa, 52242, United States
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University of California Davis Medical Center
Sacramento, California, 95817, United States
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University of Florida
Gainesville, Florida, 32610, United States
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Washington University
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.