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