Migalastat hydrochloride|cas 75172-81-5
DC Chemicals, Website: www.dcchemicals.com
Product Name: Migalastat hydrochloride|Cas Number: 75172-81-5| Catalog Number: DC10328|Other Nmaes: 1-Deoxygalactonojirimycin hydrochloride
Migalastat hydrochloride (1-Deoxygalactonojirimycin hydrochloride) is a potent and competitive inhibitor of α-galactosidase A (α-Gal A) with an IC50 of 0.04 μM for human α-Gal A.
Both IC50 and Ki values of migalastat hydrochloride toward human lysosomal a-Gal A are 0.04 μM[1].Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A. α-Gal A activity in heart, kidney, spleen, and liver is increased dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A with migalastat hydrochloride treatment. The half-life of DGJ is less than 1 day in all major issues and that of the enzyme synthesized during the DGJ treatment period is approximately 4 days[2]. Oral administration of migalastat hydrochloride reduces tissue GL-3 in fabry transgenic mice, and in urine and kidneys of some FD patients. Oral administration of migalastat hydrochloride to transgenic mice reduces elevated lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively, generally equal to or greater than observed for GL-3[3].
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