First human test: can mRNA fix broken muscles in DMD kids?
NCT ID NCT07188012
Summary
This early study is testing whether a new treatment called SPOT-mRNA03 is safe for boys with Duchenne muscular dystrophy (DMD). The treatment delivers genetic instructions to help muscles produce a missing protein called dystrophin. Six boys aged 2-6 will receive the treatment through IV infusions while researchers monitor for side effects and check if their muscles start making the needed protein.
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Contacts and locations
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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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