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Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative.
Tizzano, Eduardo F; Christie-Brown, Vanessa; Baranello, Giovanni; Germanenko, Olga; Gray, Allyson; Krstic, Marija; Lilien, Charlotte; Patel, Hinal; Servais, Laurent; Scoto, Mariacristina.
Afiliação
  • Tizzano EF; Department of Clinical and Molecular Genetics Hospital Vall d'Hebron, and Medicine Genetics Group, Vall d'Hebron Research Institute (VHIR), 08035 Barcelona, Spain.
  • Christie-Brown V; Research Programme Manager, SMA Europe e.V., Freiburg, Germany.
  • Baranello G; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Germanenko O; Patient Representative, Head of SMA Family Foundation Russia & Delegate of SMA Europe e. V., Freiburg, Germany.
  • Gray A; Paediatric Research Nurse, Great Ormond Street Hospital NHS Trust, London, United Kingdom.
  • Krstic M; Patient Representative, SMA Serbia & Delegate of SMA Europe e.V., Freiburg, Germany.
  • Lilien C; MPT: MDUK Oxford Neuromuscular Centre, Oxford, United Kingdom.
  • Patel H; The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Servais L; MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford.
  • Scoto M; The Dubowitz Neuromuscular Centre, Great Ormond Street Hospital NHS Foundation Trust, London, UK & NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London.
J Neuromuscul Dis ; 9(6): 809-820, 2022.
Article em En | MEDLINE | ID: mdl-36314215
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal Idioma: En Ano de publicação: 2022 Tipo de documento: Article