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| Product | Description | |
|---|---|---|
| Migalastat HCl | Migalastat HCl, also known as AT1001 or GR181413A, is a pharmacological chaperone that selectively binds, stabilizes, and increases cellular levels of α-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 α-galactosidase A (α-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues. Synonyms: 1,5-Dideoxy-1,5-imino-D-galactitol HCl; DGJ; (2R,3S,4R,5S)-2-methylolpiperidine-3,4,5-triol hydrochloride; Migalastat hydrochloride; Amigal. Grades: 98%. CAS No. 75172-81-5. Molecular formula: C6H13NO4.HCl. Mole weight: 199.63. |  |
| Migalastat hydrochloride | Migalastat (GR181413A free base) hydrochloride is an orally active α-galactosidase A molecular chaperone, with an IC 50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity [1]. Uses: Scientific research. Group: Signaling pathways. Alternative Names: GR181413A. CAS No. 75172-81-5. Pack Sizes: 10 mM * 1 mL; 5 mg; 10 mg; 25 mg; 50 mg; 100 mg; 250 mg. Product ID: HY-14929A. |  |
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