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Ataluren Preserves Upper Limb Function in nmDMD Patients from Study 041, a Phase 3 Placebo-Controlled Trial, and the STRIDE Registry

Author:McDonald, Craig M.   Mercuri, Eugenio   Muntoni, Francesco   Gordish-Dressman, Heather   Morgenroth, Lauren P.   Dutra de Resende, Maria Bernadete   Zhou, Shuizhen   Neeharika, Mathukumalli Lakshmi   Haginoya, Kazuhiro   Ramos-Platt, Leigh   Williams, Paula   Penematsa, Vinay   Chou, Connie   Lin, Min   Johnson, Shelley   Koladicz, Karyn   Mastrandrea, Nicholas   Werner, Christian   Trifillis, Panayiota   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.008  

Author Institution:University of California Davis School of Medicine, Davis, CA  Catholic University, Rome, Italy  UCL Institute of Child Health, London, United Kingdom  Children’s National Health System and the George Washington, Washington, DC  Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), Pittsburgh, PA  Universidade de São Paulo, São Paulo, Brazil  Children's Hospital of Fudan University & National Children Medical Center, Shanghai, China, People's Republic of  Nizam’s Institute of Medical Sciences, Hyderabad, India  Miyagi Children'S Hospital, Sendai, Japan  University of Southern California and Children's Hospital of Los Angeles, Los Angeles, CA  PTC Therapeutics Inc., South Plainfield, NJ  PTC Therapeutics Germany GmbH, Frankfurt, Germany  

Evaluation of the Biodistribution, Efficacy, and Side-Effect Profile of Deflazacort, Prednisolone and Vamorolone in a DMD Mouse Model

Author:Liu, Grace   Lipari, Phil   Mollin, Anna   Jung, Stephen   Teplova, Irina   Ma, Marcia   Li, Wencheng   Ying, Lanqing   Yeh, Shirley   Narasimhan, Jana   Trifillis, Panayiota   Leonardi, Efthimia   Welch, Ellen   Voronin, Gregory   Dong, Elaine   Mastrandrea, Nicholas   Blaize, Jonathan   Weetall, Marla   

Session Name:S34: Child Neurology and Developmental Neurology 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:S34.010  

Author Institution:PTC Therapeutics, Inc., South Plainfield, NJ  

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