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First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD.
Servais, Laurent; Eggenspieler, Damien; Poleur, Margaux; Grelet, Marc; Muntoni, Francesco; Strijbos, Paul; Annoussamy, Mélanie.
Afiliação
  • Servais L; Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK. laurent.servais@paediatrics.ox.ac.uk.
  • Eggenspieler D; Division of Child Neurology, Department of Pediatrics, Centre de Référence des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium. laurent.servais@paediatrics.ox.ac.uk.
  • Poleur M; SYSNAV, Paris, France.
  • Grelet M; Department of Neurology, Centre de Référence des Maladies Neuromusculaires, University Hospital Liège and University of Liège, Liège, Belgium.
  • Muntoni F; SYSNAV, Paris, France.
  • Strijbos P; Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, London, UK.
  • Annoussamy M; F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Nat Med ; 29(10): 2391-2392, 2023 Oct.
Article em En | MEDLINE | ID: mdl-37814063
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofia Muscular de Duchenne Limite: Humans 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 Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article