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Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study

Author:Mendell, Jerry   Sahenk, Zarife   Lehman, Kelly   Lowes, Linda   Reash, Natalie   Iammarino, Megan   Alfano, Lindsay   Lewis, Sarah   Church, Kathleen   Shell, Richard   Potter, Rachael   Griffin, Danielle   Hogan, Mark   Wang, Shufang   Mason, Stefanie   Darton, Eddie   Rodino-Klapac, Louise   

Session Name:P3: Neuromuscular Disease: Inherited Muscle Disorders 1  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P3.006  

Author Institution:Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH  Sarepta Therapeutics, Inc. , Cambridge, MA