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.
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Chemical Information
Name
Migalastat HCl
Iupac Chemical Name
Migalastat HCl
Synonyms
AT1001; AT 1001; AT-1001; GR181413A; GR 181413A; GR-181413A; 1,5-Dideoxy-1,5-imino-D-galactitol; 1-Deoxygalactonojirimycin; 1-Deoxygalactostatin; Amigal; Migalastat