A phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec for treating patients with Duchenne muscular dystrophy
Genetics in Medicine(2022)
摘要
This 3-part, Phase 2, clinical trial (NCT03769116) evaluates the safety and efficacy of systemic gene transfer of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy (DMD).
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关键词
Dystrophin
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