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              Effect of Patisiran on Polyneuropathy and Cardiomyopathy in Patients with hATTR Amyloidosis with V122I/T60A Variants: A Phase 4 Observational Study

              Author:Comenzo, Raymond L.   Shu, Francy   Malhotra, Saurabh   Hussain, Yessar   Zolty, Ronald   Desai, Urvi   Fernandez, Joel   Yureneva, Elena   Chou, Lin-Na   Capocelli, Kelley   

              Session Name:S14: General Neurology: Emerging Therapies  

              Topic:General Neurology  

              Program Number:S14.005  

              Author Institution:Tufts Medical Center, Boston, MA  UCLA Medical Center, Los Angeles, CA  Cook County Health, Chicago, IL  Austin Neuromuscular Center, Austin, TX  University of Nebraska Medical Center, Omaha, NE  Carolinas Healthcare System, Charlotte, NC  Morsani College of Medicine, Tampa, FL  Alnylam Pharmaceuticals, Cambridge, MA  

              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  

              Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD)

              Author:Proud, Crystal   Zaidman, Craig   Shieh, Perry   McDonald, Craig   Day, John   Mason, Stefanie   Guridi, Maitea   Han, Lixin   Yu, Lixi   Reid, Carol   Darton, Eddie   Wandel, Christoph   Richardson, James   Malhotra, Jyoti   Singh, Tejdip   Rodino-Klapac, Louise   Mendell, Jerry   

              Session Name:S48: Therapeutics for Muscle Disease  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:S48.006  

              Author Institution:Children’s Hospital of the King’s Daughters, Norfolk, VA  Department of Neurology, WUSTL, Washington, MO  UCLA Medical Center, Los Angeles, CA  UC Davis Health, Sacramento, CA  Department of Neurology, Stanford University, Palo Alto, 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