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Safety and Efficacy of Ataluren in nmDMD Patients from Study 041, a Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial

Author:McDonald, Craig M.   Wu, Shiwen   Gulati, Sheffali   Komaki, Hirofumi   Escobar, Rosa E.   Kostera-Pruszczyk, Anna   Vlodavets, Dmitry   Chae, Jong-Hee   Jong, Yuh-Jyh   Karachunski, Peter   Statland, Jeffrey   Lorentzos, Michelle   Penematsa, Vinay   Chou, Connie   Lin, Min   Werner, Christian   Trifillis, Panayiota   Gordon, Gregory   Koladicz, Karyn   Mastrandrea, Nicholas   Klein, Matthew   

Session Name:PL5: Clinical Trials Plenary Session  

Topic:Child Neurology and Developmental Neurology  

Program Number:PL5.001  

Author Institution:University of California Davis School of Medicine, Davis, CA  The Third Medical Center of PLA General Hospital, Beijing, China, People's Republic of  All India Institute of Medical Sciences, Ansari Nagar, India  National Center of Neurology and Psychiatry, Tokyo, Japan  National Institute of Rehabilitation, Mexico City, Mexico  Medical University of Warsaw, Warsaw, Poland  Veltischev Clinical Pediatric Research Institute of Pirogov Russian National Research Medical University, Moscow, Russian Federation  Seoul National University Hospital, Seoul, Korea, Republic of  Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung, Taiwan  University of minnesota, Minneapolis, MN  University of Kansas Medical Center, Kansas City, KS  The Children’s Hospital at Westmead, Westmead New South Wales, Australia  PTC Therapeutics Inc., South Plainfield, NJ  PTC Therapeutics Germany GmbH, Frankfurt, Germany  

FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA)

Author:Darras, Basil   Baranello, Giovanni   Boespflug-Tanguy, Odile   Day, John   Deconinck, Nicholas   Klein, Andrea   Masson, Ricardo   Mazurkiewicz-Beldzinska, Maria   Mercuri, Eugenio   Rose, Kristy   Vlodavets, Dmitry   Xiong, Hui   Zanoteli, Edmar   El-Khairi, Muna   Gerber, Marianne   Gorni, Ksenija   Kletzl, Heidemarie   Palfreeman, Laura   Dodman, Angela   Servais, Laurent   

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

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.009  

Author Institution:Department of Neurology, Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, Boston, MA  Dubowitz Neuromuscular Centre, University College London, London, UK  Developmental Neurology, Istituto Neurologico C Besta, Milan, Italy, London, United Kingdom  IMotion Institut de Myologie AP-HP, Hôpital Armand Trousseau, Paris, France  Université Paris Cité, UMR 1141, NeuroDiderot, Paris, France, Paris, France  Stanford University, Palo Alto, CA, USA, Palo Alto, CA  Neuromuscular Center, UZ Gent, Ghent, Belgium  Centre de Référence des Maladies Neuromusculaires, Children’s University Hospital, ULB, Brussels, Belgium, Ghent, Belgium  Paediatric Neurology, Uni. Children’s Hospital Basel, Basel, Switzerland  Paediatric Neurology, Inselspital, University of Bern, Bern, Switzerland, Basel, Switzerland  Developmental Neurology, Istituto Neurologico C Besta, Milan, Italy, Milan, Italy  Developmental Neurology, Medical University of Gdansk, Gdansk, Poland, Gdansk, Poland  Pediatric Neurology Institute, Catholic University, Rome, Italy, Rome, Italy  Paediatric Gait Analysis Service of New South Wales, The Children’s Hospital at Westmead, Sydney, Australia, Sydney, Australia  Children Neuromuscular Center, Pirogov Medical University, Moscow, Russia, Moscow, Russian Federation  Department of Pediatrics, Peking University First Hospital, Beijing, China, Beijing, China, People's Republic of  Department of Neurology, Faculdade de Medicina, Universidade de São Paulo, São Paulo, Brazil, Sao Paulo, Brazil  Roche Products Ltd, Welwyn Garden City, UK, Welwyn Garden City, United Kingdom  Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Basel, Switzerland  PDMA Neuro/Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Basel, Switzerland  Pharmaceutical Research, Roche Innovation Center Basel, Basel, Switzerland, Basel, Switzerland  Pharma Development Neuro, F. Hoffmann-La Roche Ltd, Basel, Switzerland, Basel, Switzerland  MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK  Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University of Liège, Liège, Belgium, Oxford, United Kingdom