Tracking a potential lifeline: 5-Year study monitors boys after groundbreaking gene therapy
NCT ID NCT06491927
Summary
This study is tracking boys for up to five years after they received an experimental gene therapy called RGX-202 for Duchenne muscular dystrophy (DMD). The main goal is to understand the long-term safety of the treatment and see if its benefits on muscle strength and movement last. No new treatment is given in this study; it only observes the 66 participants who received the therapy in a previous trial.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto, California, 94304, United States
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The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Conditions
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