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P T. 2018 May;43(5):274-309.

Pharmacotherapy of Gaucher Disease: Current and Future Options.

Abstract

The clinical manifestations of Gaucher disease, a rare genetic lysosomal storage disorder, are debilitating, and the neuronopathic forms of the disease are fatal. The authors describe the current and investigational therapies for treatment.

PMID:
29719368
PMCID:
PMC5912244

Conflict of interest statement

Disclosures: The authors report no commercial or financial interests in regard to this article.

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