10-Year watch: tracking boys after groundbreaking gene therapy
NCT ID NCT05689164
Summary
This study follows boys with Duchenne muscular dystrophy for 10 years after they received an experimental gene therapy called fordadistrogene movaparvovec in earlier Pfizer trials. The main goal is to monitor long-term safety and see how well the treatment continues to work over time. Participants will have annual check-ins to track their health, muscle function, and heart health.
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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
Explore the condition pages connected to this study.