Gene therapy trial aims to slow duchenne muscular dystrophy
NCT ID NCT03368742
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 23 times
Summary
This early-stage trial tests a single dose of a gene therapy called SGT-001 in 12 boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Participants will be followed for about 5 years to monitor side effects and any changes in their condition.
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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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