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              EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics

              Author:Muntoni, Francesco   Mercuri, Eugenio   Schara Schmidt, Ulrike   Komaki, Hirofumi   Richardson, James   Singh, Tejdip   Guridi, Maitea   Mason, Stefanie   Murphy, Alex   Yu, Lixi   Reid, Carol   Darton, Eddie   Wandel, Christoph   Mendell, Jerry   

              Session Name:P5: Neuromuscular Disease: Inherited Muscle Disorders 2  

              Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

              Program Number:P5.012  

              Author Institution:The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust, London, United Kingdom  Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy  Department of Pediatric Neurology, Center for Neuromuscular Disorders in Children and Adolescents, University Clinic Essen, University of Duisburg-Essen, Essen, Germany  Translational Medical Center, National Center of Neurology and Psychiatry, Kodaira, Japan  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  

              JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam

              Author:Chiriboga, Claudia   Bruno, Claudio   Duong, Tina   Fischer, Dirk   Kirschner, Janbernd   Scoto, Mariacristina   Mercuri, Eugenio   Gerber, Marianne   Gorni, Ksenija   Kletzl, Heidemarie   Carruthers, Imogen   Martin, Carmen   Gidaro, Teresa   Muntoni, Francesco   

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

              Topic:Child Neurology and Developmental Neurology  

              Program Number:P7.004  

              Author Institution:Columbia Irving Medical Center, New York, NY  Istituto Giannina Gaslini, Genoa, Italy  Stanford University, Palo Alto, CA  University Children’s Hospital Basel, Basel, Switzerland  University of Freiburg, Freiburg, Germany  Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital Trust, London, United Kingdom  Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy  F. Hoffmann-La Roche Ltd, Basel, Switzerland  Roche Innovation Center Basel, Basel, Switzerland  Roche Products Ltd, Welwyn Garden City, United Kingdom  Hoffmann-La Roche Ltd, Basel, Switzerland