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Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory,randomised, placebo-controlled phase 2 study
Authors:Prof Thomas Voit  Prof Haluk Topaloglu  Prof Volker Straub  Prof Francesco Muntoni  Nicolas Deconinck  Giles Campion  Sjef J De Kimpe  Michelle Eagle  Michela Guglieri  Steve Hood  Lia Liefaard  Afrodite Lourbakos  Allison Morgan  Joanna Nakielny  Naashika Quarcoo  Valeria Ricotti  Katie Rolfe  Laurent Servais  Claire Wardell  Rosamund Wilson  Padraig Wright  John E Kraus
Institution:1. Institut de Myologie, Universite Pierre et Marie Curie, GH Pitié-Salpêtrière, UPMC-INSERM UMR 974, Paris, France;2. Department of Pediatrics, Hacettepe University, Ankara, Turkey;3. Institute of Genetic Medicine, University of Newcastle, Newcastle, UK;4. The Dubowitz Neuromuscular Centre, University College London Institute of Child Health, London, UK;5. Department of Neurology, Universitair Ziekenhuis Gent, Ghent, Belgium;6. Department of Paediatric Neurology, Hopital des Enfants reine Fabiola, ULB, Belgium;g Prosensa Therapeutics BV, Leiden, Netherlands;h Methis Clinical, Ascot, Berkshire, UK;i GlaxoSmithKline, London, UK;j Takeda, London, UK;k GlaxoSmithKline, Stevenage, UK;l Spica Consultants Ltd, Marlborough, UK;m GlaxoSmithKline, Raleigh-Durham, NC, USA
Abstract:
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