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Autor:
Straub V; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Balabanov P; European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Management Department, CNS and Ophthalmology Office, London, UK., Bushby K; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Ensini M; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Goemans N; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium., De Luca A; Unit of Pharmacology, Department of Pharmacy-Drug Sciences, University of Bari, Bari, Italy., Pereda A; Duchenne Parent Project, Madrid, Spain., Hemmings R; Medicines and Healthcare Product Regulatory Agency, London, UK., Campion G; BioMarin Pharmaceutical, Developmental Sciences, Novato, CA, USA., Kaye E; Sarepta Therapeutics, Cambridge, MA, USA., Arechavala-Gomeza V; Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Spain., Goyenvalle A; Université de Versailles Saint Quentin en Yvelines, Inserm U1179, Montigny le Bretonneux, France., Niks E; Leiden University Medical Center, Leiden, Netherlands., Veldhuizen O; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK., Furlong P; Parent Project Muscular Dystrophy, Hackensack, NJ, USA., Stoyanova-Beninska V; Medicines Evaluation Board, Utrecht, Netherlands., Wood MJ; Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, UK., Johnson A; Joining Jack, Wigan, UK., Mercuri E; Pediatric Neurology, Catholic University, Rome, Italy., Muntoni F; Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases, University College London Institute of Child Health and Great Ormond Street Hospital, London, UK., Sepodes B; Faculdade de Farmácia, Universidade de Lisboa, Lisbon, Portugal., Haas M; European Medicines Agency, Human Medicines Evaluation Division, Scientific and Regulatory Management Department, CNS and Ophthalmology Office, London, UK., Vroom E; United Parent Projects Muscular Dystrophy, Amsterdam, Netherlands., Aartsma-Rus A; John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK; Leiden University Medical Center, Leiden, Netherlands. Electronic address: a.m.rus@lumc.nl.
Publikováno v:
The Lancet. Neurology [Lancet Neurol] 2016 Jul; Vol. 15 (8), pp. 882-890.