Experimental gene therapy targets duchenne MD in young boys
NCT ID NCT05429372
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated early, so full results are not available.
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 (a gene therapy)
What this could lead to
If successful, this gene therapy could help boys with Duchenne muscular dystrophy produce a shortened form of dystrophin, potentially slowing muscle damage.
What could go wrong
This early-phase trial was terminated, so results are limited. Gene therapies can cause immune reactions or other serious side effects, and long-term benefits are unproven.
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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CTSI Clinical Research Center
Salt Lake City, Utah, 84108, United States
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Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
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The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
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UF Health Shands Hospital
Gainesville, Florida, 32610, United States
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University of Florida
Gainesville, Florida, 32610, 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 Hospital
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital & Clinics Investigational Drug Services
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