Gene therapy watch: 10-Year safety check for duchenne patients
NCT ID NCT05689164
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 26 times
Summary
This study checks the long-term safety and effects of an experimental gene therapy called fordadistrogene movaparvovec in people with Duchenne muscular dystrophy who received it in a previous Pfizer study. Participants will be followed for up to 10 years with annual visits. The goal is to monitor side effects and track changes in muscle function over time.
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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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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
Conditions
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