Gene therapy trial aims to slow duchenne muscular dystrophy in boys
NCT ID NCT03368742
First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 4 times
Summary
This study tests a single dose of SGT-001 gene therapy in 12 boys (children and teens) with Duchenne muscular dystrophy. The main goal is to check safety and how well the body tolerates the treatment. Participants will be followed for about 5 years to monitor side effects and any changes in muscle function.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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
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Other studies related to the condition(s) this trial covers.
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