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              One-year Data from ENDEAVOR, a Phase 1b Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD)

              Author:Zaidman, Craig   Proud, Crystal   McDonald, Craig   Mason, Stefanie   Guridi, Maitea   Wang, Shufang   Reid, Carol   Darton, Eddie   Wandel, Christoph   Lewis, Sarah   Malhotra, Jyoti   Griffin, Danielle   Potter, Rachael   Rodino-Klapac, Louise   Mendell, Jerry   

              Session Name:S48: Therapeutics for Muscle Disease  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S48.003  

              Author Institution:Department of Neurology, WUSTL, Washington, MO  Children’s Hospital of the King’s Daughters, Norfolk, VA  UC Davis Health, Sacramento, CA  Sarepta Therapeutics, Inc. , Cambridge, MA  F. Hoffmann-La Roche Ltd, Basel, Switzerland  Roche Products Ltd, Welwyn Garden City, United Kingdom  Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH  

              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  

              PK/PD Modeling to Inform Clinical Development of an Adeno-associated Virus Gene Transfer Therapy for Duchenne Muscular Dystrophy

              Author:East, Lilly   Potter, Rachael   Snedeker, John   Haile, Alex   Wier, Chris   Rodino-Klapac, Louise   

              Session Name:S48: Therapeutics for Muscle Disease  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S48.005  

              Author Institution:Sarepta Therapeutics, Inc. , Cambridge, MA  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  

              Evaluating Pharmacology and Efficacy of Delandistrogene Moxeparvovec in Young and Aged DMDmdx Rats

              Author:Potter, Rachael   Wier, Chris   Cooper-Olson, Grace   Wheeler, Esther   Anderbery, Emily   Kempton, Amber   Clements, Leah   Adegboye, Kaitlin   Haile, Alex   Peterson, Ellyn   Rodino-Klapac, Louise   

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

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:P3.008  

              Author Institution:Sarepta Therapeutics, Inc. , Cambridge, MA  Sarepta Therapeutics, Inc., Cambridge, MA