Rapid preparation of (3R,4S,5R) polyhydroxylated pyrrolidine-based libraries to discover a pharmacological chaperone for treatment of Fabry disease.
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DOI: 10.1016/j.ejmech.2016.10.004
Abstract: The rapid discovery of a pharmacological chaperone toward human α-Gal A for the treatment of Fabry disease is described. Two polyhydroxylated pyrrolidines with the (3R,4S,5R) configuration pattern underwent rapid substituent diversity by conjugating the primary… read more here.
Keywords: pharmacological chaperone; treatment fabry; fabry disease;
Treatment of Fabry Disease: Established and Emerging Therapies
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DOI: 10.3390/ph16020320
Abstract: Fabry disease (FD) is a rare, X-linked inherited disorder of glycosphingolipid metabolism. It leads to the progressive accumulation of globotriaosylceramide within lysosomes due to a deficiency of α-galactosidase A enzyme. It involves multiple organs, predominantly… read more here.
Keywords: established emerging; disease established; treatment fabry; treatment ... See more keywords