Gene therapy SPOT-03 hopes to restore muscle protein in boys with DMD
NCT ID NCT07188012
First seen Sep 30, 2025 · Last updated May 17, 2026 · Updated 23 times
Summary
This early-phase study tests a new gene therapy called SPOT-03 in 9 boys aged 2 to 8 with Duchenne muscular dystrophy (DMD). The main goal is to see if the treatment is safe and tolerable. Researchers will also check if SPOT-03 can increase dystrophin, a key muscle protein missing in DMD, and measure changes in muscle and fat tissue.
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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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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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