Gene therapy trial hopes to restore muscle protein in duchenne patients
NCT ID NCT04626674
First seen Jan 04, 2026 · Last updated May 22, 2026 · Updated 22 times
Summary
This study tests a gene therapy called delandistrogene moxeparvovec (SRP-9001) in people with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and can help the body produce dystrophin, a protein missing in DMD. The trial includes children and adults, both able to walk and not. Participants will be followed for up to 156 weeks.
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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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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
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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
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Stanford University
RECRUITINGPalo Alto, California, 94304, United States
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University of California, Davis
RECRUITINGSacramento, California, 95616, United States
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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