New gene therapy trial hopes to restore muscle protein in duchenne boys
NCT ID NCT04626674
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (Cohort 8) and will follow them for up to 156 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
delandistrogene moxeparvovec (gene therapy)
What this could lead to
If successful, this could point toward a treatment that helps boys with Duchenne produce a key muscle protein, potentially slowing disease progression.
What could go wrong
This is an early Phase 1 study, so safety and effectiveness are not yet proven. Risks include liver injury and immune reactions, and long-term benefits are uncertain.
Disclaimer
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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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of The King's Daughters
RECRUITINGNorfolk, Virginia, 23507, United States
Contact Email: •••••@•••••
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Nationwide Children's Hospital
ACTIVE_NOT_RECRUITINGColumbus, Ohio, 43205, United States
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Neurology Rare Disease Center
RECRUITINGFlower Mound, Texas, 75028, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
Contact Email: •••••@•••••
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University of California, Davis
RECRUITINGSacramento, California, 95616, United States
Contact
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact