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              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