75172-81-5
基本信息
脫氧半乳糖野生霉素 鹽酸鹽
Amigal
Migalastat HCl
Unii-cly7m0xd20
GALACTOSTATIN HCL
DGJ, HYDROCHLORIDE
Migalastat hydrochloride
Galactostatin hydrochloride
DEOXYGALACTONOJIRIMYCIN HCL
1-DEOXYGALACTONOJIRIMYCIN HCL
安全數(shù)據(jù)
常見問題列表
脫氧半乳糖野生霉素鹽酸鹽是一種有效和競爭性的α-galactosidase A抑制劑,對人α-Gal A的 IC50值為0.04 μM。
IC50: 0.04 μM (human α-Gal A); Ki: 0.04 μM (human α-Gal A)
Both IC 50 and K i values of Migalastat (GR181413A) hydrochloride toward human lysosomal a-Gal A are 0.04 μM.
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 (GR181413A) 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. Oral administration of Migalastat (GR181413A) hydrochloride reduces tissue GL-3 in fabry transgenic mice, and in urine and kidneys of some FD patients. Oral administration of Migalastat (GR181413A) 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.