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

Haematologica. 2020 May;105(5):e206-e209. doi: 10.3324/haematol.2019.224717. Epub 2019 Sep 26.
No abstract available

Publication types

  • Letter

MeSH terms

  • Drug Development
  • Gaucher Disease* / drug therapy
  • Glucosylceramidase* / genetics
  • Humans
  • Macrophages

Substances

  • Glucosylceramidase