Gene therapy SPOT-03 aims to rebuild muscle protein in boys with duchenne
NCT ID NCT07188012
First seen Sep 30, 2025 · Last updated May 20, 2026 · Updated 24 times
Summary
This early-phase study tests a gene therapy called SPOT-03 in 9 boys with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Researchers will also check if the therapy increases dystrophin protein levels in muscles, which is missing in DMD. The study is currently recruiting boys aged 2 to under 8 years who can walk at least 10 meters.
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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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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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