First human test of new gene therapy for devastating muscle disease
NCT ID NCT06114056
Summary
This early-stage study is testing the safety and initial effects of a single dose of an experimental gene therapy called JWK007 in boys with Duchenne muscular dystrophy (DMD). The trial will enroll only 3 boys aged 5-10 years to receive a low-dose injection. The main goal is to see if the treatment is safe and well-tolerated, while also checking for any early signs of improvement in muscle function.
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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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