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P314: Long-term Safety and Sustained Functional Benefit in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec: A Phase 1/2a Study*

Erin O’Rourke,Jerry R. Mendell,Zarife Sahenk,Kelly J. Lehman,Linda P. Lowes,Natalie F. Reash,Megan Iammarino,Lindsay Alfano,Sarah Lewis,Kathleen Church,Richard Shell,Rachael A. Potter,Danielle Griffin,Mark J. Hogan,Shufang Wang,Stefanie Mason,Eddie Darton,Louise Rodino-Klapac

Genetics in Medicine Open（2023）

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