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Functional assessment of glucocerebrosidase modulator efficacy in primary patient-derived macrophages is essential for drug development and patient stratification.
Welsh, Natalie J; Gewinner, Christina A; Mistry, Kavita; Koglin, Mumta; Cooke, Juniebel; Butler, Matthew; Powney, Ben; Roberts, Malcolm; Staddon, James M; Schapira, Anthony H V.
Afiliação
  • Welsh NJ; Eisai-UCL Drug Discovery and Development Collaboration, UCL Queen Square Institute of Neurology, School of Life Sciences and Medicine, University College London, London.
  • Gewinner CA; Eisai-UCL Drug Discovery and Development Collaboration, UCL Queen Square Institute of Neurology, School of Life Sciences and Medicine, University College London, London.
  • Mistry K; Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire.
  • Koglin M; Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire.
  • Cooke J; Lysosomal Storage Disorders Unit, Royal Free Hospital, London.
  • Butler M; Eisai-UCL Drug Discovery and Development Collaboration, UCL Queen Square Institute of Neurology, School of Life Sciences and Medicine, University College London, London.
  • Powney B; Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire.
  • Roberts M; Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire.
  • Staddon JM; Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire.
  • Schapira AHV; Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London, UK a.schapira@ucl.ac.uk.
Haematologica ; 105(5): e206-e209, 2020 05.
Article em En | MEDLINE | ID: mdl-31558665

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Gaucher / Glucosilceramidase Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Gaucher / Glucosilceramidase Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article