One-shot gene therapy aims to give duchenne boys stronger muscles
NCT ID NCT07160634
First seen Oct 31, 2025 · Last updated May 20, 2026 · Updated 33 times
Summary
This study tests a single intravenous dose of SGT-003, a gene therapy, in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it improves muscle function, like how fast they can stand up or climb stairs. Participants will receive either the therapy or a placebo first, then switch, and be followed for at least 5 years.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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BC Children's Hospital
RECRUITINGVancouver, British Columbia, Canada
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Children's Hospital of Westmead
RECRUITINGSydney, New South Wales, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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