Gene therapy for duchenne MD shows promise but study halted early
NCT ID NCT03362502
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 29 times
Summary
This study tested a single dose of a gene therapy called PF-06939926 in 23 people with Duchenne muscular dystrophy (DMD), both those who could still walk and those who could not. The main goal was to check safety and side effects over one year. The trial was stopped early, but researchers looked at how well the therapy was tolerated and measured changes in muscle strength and function.
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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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Locations
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Biospecimen Repository & Processing Core - BPRC
Durham, North Carolina, 27710, United States
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CCTS Clinical Research Center
Salt Lake City, Utah, 84108, United States
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Duke Cardiovascular Magnetic Resonance Center
Durham, North Carolina, 27710, United States
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Duke Children's Hospital & Health Center
Durham, North Carolina, 27710, United States
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Duke Neurology
Durham, North Carolina, 27705, United States
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Duke University Hospital Investigational Drug Services (IDS) Pharmacy
Durham, North Carolina, 27710, United States
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Duke University Medical Center, Lenox Baker Children's Hospital
Durham, North Carolina, 27705, United States
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MRI Research Center
Los Angeles, California, 90095, 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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Ronald Reagan UCLA Medical Center (Investigational Drug Section)
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center - Interventional Radiology
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center Drug Information Center
Los Angeles, California, 90095, United States
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UCLA (David Geffen School of Medicine)
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 Mattel Children's Hospital
Los Angeles, California, 90095, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UCLA Outpatient Surgery 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 Hospital
Salt Lake City, Utah, 84112, 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
Conditions
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