Journal
EUROPEAN JOURNAL OF PHARMACOLOGY
Volume 926, Issue -, Pages -Publisher
ELSEVIER
DOI: 10.1016/j.ejphar.2022.175023
Keywords
Gaucher disease; Enzyme replacement therapy; Substrate reduction therapy; Gene therapy; Hematopoietic stem cell transplantation; Genome editing
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This paper provides an update on approved therapies and potential curative therapies for Gaucher disease (GD), the most common lysosomal disorder.
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.
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