Gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT03368742
Summary
This early-stage study is testing a single dose of an experimental gene therapy called SGT-001 in boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and well-tolerated. Researchers will also check if the therapy helps produce a needed muscle protein and improves muscle strength and function over five years of follow-up.
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Contacts and locations
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Locations
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David Geffen School of Medicine at UCLA
Los Angeles, California, 90095, United States
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University of Florida
Gainesville, Florida, 32610, United States
Conditions
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