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Pharmacologic chaperoning as a strategy to treat Gaucher disease
Author(s) -
Yu Zhanqian,
Sawkar Anu R.,
Kelly Jeffery W.
Publication year - 2007
Publication title -
the febs journal
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.981
H-Index - 204
eISSN - 1742-4658
pISSN - 1742-464X
DOI - 10.1111/j.1742-4658.2007.06042.x
Subject(s) - disease , lysosomal storage disorders , food and drug administration , glucocerebrosidase , intensive care medicine , gaucher's disease , medicine , chaperone (clinical) , pharmacology , pathology
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.