A phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec for treating patients with Duchenne muscular dystrophy

Erin O'Rourke, Jerry Mendell, Perry Shieh, Zarife Sahenk, Kelly Lehman, Linda Lowes, Natalie Reash, Megan Iammarino, Lindsay Alfano, Brenna Powers, Jeremy Woods, Christy Skura, Howard Mao, Loretta Staudt, Rachael Potter, Danielle Griffin, Sarah Lewis, Sameer Upadhyay, Teji Singh, Louise Rodino-Klapac

Genetics in Medicine（2022）

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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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Dystrophin

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