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Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.
Servais, Laurent; Mercuri, Eugenio; Straub, Volker; Guglieri, Michela; Seferian, Andreea M; Scoto, Mariacristina; Leone, Daniela; Koenig, Erica; Khan, Navid; Dugar, Ashish; Wang, Xiaodong; Han, Baoguang; Wang, Dan; Muntoni, Francesco.
Afiliação
  • Servais L; I-Motion Institute, Hôpital Armand Trousseau, Paris, France.
  • Mercuri E; Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège & University of Liège, Liège, Belgium.
  • Straub V; MDUK Oxford Neuromuscular Centre, University of Oxford, Oxford, United Kingdom.
  • Guglieri M; Pediatric Neurology Unit, Università Cattolica del Sacro Cuore Roma, Rome, Italy.
  • Seferian AM; Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
  • Scoto M; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
  • Leone D; John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
  • Koenig E; I-Motion Institute, Hôpital Armand Trousseau, Paris, France.
  • Khan N; Dubowitz Neuromuscular Centre, University College London, Great Ormond Street Institute of Child Health, London, United Kingdom.
  • Dugar A; National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom.
  • Wang X; Nemo Clinical Centre, Fondazione Policlinico Universitario A Gemelli IRCCS, Rome, Italy.
  • Han B; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Wang D; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
  • Muntoni F; Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA.
Nucleic Acid Ther ; 32(1): 29-39, 2022 02.
Article em En | MEDLINE | ID: mdl-34788571
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article