Migalastat HCl, also known as AT1001 or GR181413A, is a pharmacological chaperone that selectively binds, stabilizes, and increases cellular levels of -Gal A. Oral administration of migalastat HCl reduces tissue GL-3 in Fabry transgenic mice, and in urine and kidneys of some FD patients. Migalastat HCl may provide a potential novel genotype-specific treatment for Fabry Disease (FD). Phase 3 studies are ongoing. Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme -galactosidase A (-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues.