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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