Update of treatment for Gaucher disease

Eur J Pharmacol. 2022 Jul 5:926:175023. doi: 10.1016/j.ejphar.2022.175023. Epub 2022 May 13.


Gaucher disease (GD), the most common lysosomal disorders, is a rare autosomal recessive hereditary disease that is caused by deficiency of glucosylceramidase. For now, there are five approved therapies for GD, which are used to treat thousands of patients with GD. Despite success of approved therapies, many unresolved issues attract academic institutions and industry to develop potential therapies to resolve them. This paper updated the latest information about approved therapies and potential curative therapies.

Keywords: Enzyme replacement therapy; Gaucher disease; Gene therapy; Genome editing; Hematopoietic stem cell transplantation; Substrate reduction therapy.

Publication types

  • Review

MeSH terms

  • Enzyme Replacement Therapy
  • Gaucher Disease* / drug therapy
  • Humans
  • Lysosomes