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Effectiveness of Ataluren in Patients with nmDMD: Confirmatory Evidence from the STRIDE Registry

Author:Werner, Christian   Mercuri, Eugenio   Buccella, Filippo   Nascimento Osorio, Andrés   Tulinius, Már   Desguerre, Isabelle   Dutra de Resende, Maria Bernadete   McDonald, Craig   Gordish-Dressman, Heather   Morgenroth, Lauren   Johnson, Shelley   Krolick, Alexis   Anbu, Balaji   L,   

Session Name:P7: Child Neurology and Developmental Neurology: Neuromuscular 2  

Topic:Child Neurology and Developmental Neurology  

Program Number:P7.008  

Author Institution:PTC Therapeutics Germany GmbH, Frankfurt, Germany  Catholic University, Rome, Italy  Parent Project APS Italy, Rome, Italy  Unidad de Patología Neuromuscular, Hospital Sant Joan de Déu, Universidad de Barcelona, Barcelona, Spain  Queen Silvia Children’s Hospital, University of Gothenburg, Gothenburg, Sweden  Hôpital Necker – Enfants Malades, Paris, France  University of São Paulo, São Paulo, Brazil  University of California Davis School of Medicine, Davis, CA  Children's National Health System and the George Washington, Washington DC, WA  Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), Pittsburgh, PA  PTC Therapeutics Inc., Warren, NJ  University College London Great Ormond Street Institute of Child Health, London, United Kingdom  

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