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Review
. 2007 Oct;274(19):4944-50.
doi: 10.1111/j.1742-4658.2007.06042.x.

Pharmacologic chaperoning as a strategy to treat Gaucher disease

Zhanqian Yu  1 , Anu R Sawkar, Jeffery W Kelly
Affiliations
Free article
Review

Pharmacologic chaperoning as a strategy to treat Gaucher disease

Zhanqian Yu et al. FEBS J. 2007 Oct.
Free article

Abstract

We briefly introduce the most common lysosomal storage disorder, Gaucher disease, concisely describe the Food and Drug Administration approved strategies to ameliorate Gaucher disease, and then outline the emerging pharmacologic chaperone strategy that offers the promise to remedy this malady.

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