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  

Results from the first four regimens of the HEALEY ALS Platform Trial

Author:Paganoni, Sabrina   Berry, James   Quintana, Melanie   Macklin, Eric   Saville, Benjamin   Andrews, Jinsy   Shefner, Jeremy   Fournier, Christina   Babu, Suma   Maragakis, Nicholas   Oskarsson, Bjorn   Detry, Michelle   Chase, Marianne   Sherman, Alex   Yu, Hong   Pothier, Lindsay   Drake, Kristin   Chibnik, Lori   Bind, Marie-Abele   Vestrucci, Matteo   McGlothlin, Anna   Marion, Joseph   Duda, Petra   Harvey, Brittany   Qureshi, Irfan   Donohue, Mary   Granit, Volkan   Grossman, Katheryn   Glanzman, Robert   Hotchkin, Michael   Goldberg, Y Paul   Leitner, Melanie   Hayden, Michael   Cudkowicz, Merit   

Session Name:PL5: Clinical Trials Plenary Session  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:PL5.004  

Author Institution:Massachusetts General Hospital, Boston, MA  Berry Consultants, Austin, TX  MGH Biostatistics Center, Boston, MA  Columbia University Medical Center, New York, NY  Barrow Neurological Institute, Phoenix, AZ  Emory University, Atlanta, GA  Johns Hopkins University School of Medicine, Baltimore, MD  Mayo Clinic, Jacksonville, FL  Harvard School of Public Health, Boston, MA  UCB, Brussels, Belgium  Biohaven Pharmaceuticals, New Haven, CT  Biohaven Pharmaceuticals, Inc., New Haven, CT  Biohaven Pharmaceuticals, East Petersburg, PA  Clene Nanomedicine Inc, Holladay, UT  Clene Nanomedicine, Inc., Salt Lake City, UT  Prilenia Therapeutics, Herzliya, Israel  Accelerating Neuroventures, Boston, MA