Migalastat HCl Datasheet DC Chemicals

Description	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.
Cat.No	DC75368
Name	Migalastat HCl

Chemical Properties

CAS	75172-81-5
Formula	C6H13NO4
MW	163.17
Storage	2 years -20°C Powder, 2 weeks 4°C in DMSO, 6 months -80°C in DMSO

References

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