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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping.
Mercuri, E; Seferian, A M; Servais, L; Deconinck, N; Stevenson, H; Ni, X; Zhang, W; East, L; Yonren, S; Muntoni, F.
Afiliação
  • Mercuri E; Pediatric Neurology Unit, Università Cattolica del Sacro Cuore Roma, Rome, Italy; Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
  • Seferian AM; I-Motion Institute, Hôpital Armand Trousseau, Paris, France.
  • Servais L; 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 R
  • Deconinck N; 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.
  • Stevenson H; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Ni X; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Zhang W; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • East L; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Yonren S; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Muntoni F; 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.
Neuromuscul Disord ; 33(6): 476-483, 2023 06.
Article em En | MEDLINE | ID: mdl-37207382
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
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XML
PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Idioma: En Ano de publicação: 2023 Tipo de documento: Article