Study Summary
This study is a single-center, single-arm, non-randomized, open-label, non-controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK007 injection in pediatric patients with Duchenne Muscular Dystrophy (DMD).
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JWK007 Single intravenous infusion administrationBIOLOGICAL
Each patient receives a single intravenous infusion of JWK007 at a dose of 1.0 × 10\^14 vg/kg.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| West China Hospital, Sichuan University | Chengdu | Sichuan | China |