First test of new gene therapy for devastating muscle disease in children
NCT ID NCT06114056
Summary
This early-stage study is testing the safety and initial effects of a single intravenous gene therapy called JWK007 in three young boys with Duchenne muscular dystrophy (DMD). The therapy aims to deliver a working copy of a crucial muscle gene that these boys are missing. Researchers will monitor for side effects and check if the treatment helps the boys produce a needed muscle protein and improve their movement abilities.
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Contacts and locations
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Locations
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West China Hospital, Sichuan University
Chengdu, Sichuan, 610041, China
Conditions
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