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Functional assessment of glucocerebrosidase modulator efficacy in primary patient-derived macrophages is essential for drug development and patient stratification

Eisai-UCL Drug Discovery and Development Collaboration, UCL Queen Square Institute of Neurology, School of Life Sciences and Medicine, University College London, London
Eisai-UCL Drug Discovery and Development Collaboration, UCL Queen Square Institute of Neurology, School of Life Sciences and Medicine, University College London, London
Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire
Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire
Lysosomal Storage Disorders Unit, Royal Free Hospital, London
Eisai-UCL Drug Discovery and Development Collaboration, UCL Queen Square Institute of Neurology, School of Life Sciences and Medicine, University College London, London
Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire
Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire
Neurology Innovation Centre, Hatfield Research Laboratories, Eisai Ltd, Hatfield, Hertfordshire
Department of Clinical and Movement Neurosciences, UCL Queen Square Institute of Neurology, University College London, London, UK
Vol. 105 No. 5 (2020): May, 2020 https://doi.org/10.3324/haematol.2019.224717