首页> Neuromuscular disorders : NMD> Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.

Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.

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第一作者： E,Mercuri

第一单位： Pediatric Neurology Unit, Università Cattolica del Sacro Cuore Roma, Rome, Italy; Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy.

作者： E,Mercuri ^[1] ; A M,Seferian ^[2] ; L,Servais ^[3] ; N,Deconinck ^[4] ; H,Stevenson ^[5] ; X,Ni ^[5] ; W,Zhang ^[5] ; L,East ^[5] ; S,Yonren ^[5] ; F,Muntoni ^[6] ; 4658-102 Study Group

作者单位： Pediatric Neurology Unit, Università Cattolica del Sacro Cuore Roma, Rome, Italy; Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy. ^[1] I-Motion Institute, Hôpital Armand Trousseau, Paris, France. ^[2] I-Motion Institute, Hôpital Armand Trousseau, Paris, France; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium; MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK. ^[3] Neuromuscular Reference Center, UZ Gent, Ghent, Belgium; Centre de Référence Neuromusculaire and Paediatric Neurology Department, Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, 1020 Brussels, Belgium. ^[4] Sarepta Therapeutics, Inc., Cambridge, MA, USA. ^[5] Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, UK; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK. Electronic address: f.muntoni@ucl.ac.uk. ^[6]

关键词 Clinical trial Duchenne muscular dystrophy Eteplirsen Safety