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Gene Therapy with Bidridistrogene Xeboparvovec for Limb-Girdle Muscular Dystrophy Type 2E/R4: Phase 1/2 Trial Results

Jerry R. Mendell,Eric R. Pozsgai,Sarah Lewis,Danielle A. Griffin,Linda P. Lowes,Lindsay N. Alfano,Kelly J. Lehman,Kathleen Church,Natalie F. Reash,Megan A. Iammarino,Brenna Sabo,Rachael Potter,Sarah Neuhaus,Xiaoxi Li,Herb Stevenson,Louise R. Rodino-Klapac

NATURE MEDICINE（2024）

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Key words
Gene Therapy
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