Real-World data sought on DMD Exon-Skipping drugs
NCT ID NCT06606340
First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This observational study will follow 300 people with Duchenne muscular dystrophy who are already taking exon-skipping therapies (eteplirsen, golodirsen, or casimersen) as part of their routine care. Researchers will collect data on movement, lung function, and heart function over time to see how these treatments work in everyday practice. No new drugs are being tested; the goal is to gather long-term safety and effectiveness information.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
eteplirsen, golodirsen, casimersen
What this could lead to
If successful, this study could provide real-world evidence on how well these exon-skipping therapies work over the long term for Duchenne muscular dystrophy.
What could go wrong
This is an observational study, not a controlled trial, so results may be influenced by factors like differences in patient care. It does not test new treatments, only monitors existing ones.
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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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