Gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT03368742
First seen Nov 01, 2025 · Last updated Apr 16, 2026 · Updated 20 times
Summary
This early-stage study is testing a single intravenous 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 tolerable. Researchers will also look for early signs that it might help by measuring a key muscle protein and checking muscle and heart function over five years of follow-up.
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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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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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