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              Bulbar Function in Children with Two or Three SMN2 Copies Who Received Onasemnogene Abeparvovec Presymptomatically for Spinal Muscular Atrophy

              Author:McGrattan, Katlyn   Shell, Richard   Hurst-Davis, Rebecca   Dunaway Young, Sally   Baranello, Giovanni   Lavrov, Arseniy   O'Brien, Eamonn   Wallach, Shiri   LaMarca, Nicole   Reyna, Sandra P.   Darras, Basil   

              Session Name:P7: Child Neurology and Developmental Neurology: Spinomuscular Atrophy/Muscular Dystrophies  

              Topic:Child Neurology and Developmental Neurology  

              Program Number:P7.008  

              Author Institution:University of minnesota, Minneapolis, MN  Nationwide Children’s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH  Primary Children’s Hospital, Salt Lake City, UT  Stanford University School of Medicine, Stanford, CA  The Dubowitz Neuromuscular Centre, Developmental Neuroscience Research and Teaching Department, UCL Great Ormond Street Institute of Child Health and NIHR Great Ormond Street Hospital Biomedical Research Centre & Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom  Novartis Gene Therapies, Inc., Cambridge, United Kingdom  Novartis, Dublin, Ireland  Novartis Gene Therapies, Inc., Bannockburn, IL  Boston Children’s Hospital, Harvard Medical School, Boston, MA