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              A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD)

              Author:Mendell, Jerry   Shieh, Perry   Sahenk, Zarife   Lehman, Kelly   Lowes, Linda   Reash, Natalie   Iammarino, Megan   Alfano, Lindsay   Sabo, Brenna   Woods, Jeremy   Skura, Christy   Mao, Howard   Staudt, Loretta   Potter, Rachael   Griffin, Danielle   Lewis, Sarah   Wang, Shufang   Singh, Tejdip   Rodino-Klapac, Louise   

              Session Name:S48: Therapeutics for Muscle Disease  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S48.004  

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

              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