One-shot gene therapy aims to help boys with duchenne walk and climb stairs
NCT ID NCT07160634
First seen Oct 31, 2025 · Last updated May 15, 2026 · Updated 32 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 their ability to stand up, walk, and climb stairs compared to a placebo. All participants will receive the therapy eventually, and they will be followed for at least 5 years.
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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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