Gene therapy for duchenne muscular dystrophy under Long-Term watch
NCT ID NCT05689164
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. The goal is to better understand the therapy's long-term safety and effectiveness.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
fordadistrogene movaparvovec (gene therapy)
What this could lead to
If successful, this study could confirm the long-term safety and lasting benefits of this gene therapy for Duchenne muscular dystrophy.
What could go wrong
This is a small, terminated follow-up study with only 7 participants, so results may not apply widely. Gene therapy carries risks like immune reactions or unknown long-term side effects.
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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Duke Children's Health Center
Durham, North Carolina, 27710, United States
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Duke Lenox Baker Children's
Durham, North Carolina, 27705, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Reed Neurological Research Center
Los Angeles, California, 90095, United States
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UCLA Children's Heart Center
Los Angeles, California, 90095, United States
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UCLA Clinical Lab Services
Los Angeles, California, 90095, United States
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UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)
Los Angeles, California, 90095, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132, United States
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University of Utah Craig H. Neilsen Rehabilitation Hospital
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital
Salt Lake City, Utah, 84112, United States
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University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108, United States