Migalastat hydrochloride (GR181413A) 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 K_i values of Migalastat hydrochloride (GR181413A) toward human lysosomal a-Gal A are 0.04 μM.

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A.
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM).
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment.
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively.

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A.
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM).
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment.
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively.

西域 has not independently confirmed the accuracy of these methods. They are for reference only.

• . Asano N, et al. In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives. Eur J Biochem. 2000 Jul;267(13):4179-86.  [Content Brief]

  . Ishii S, et al. Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease. J Pharmacol Exp Ther. 2009 Mar;328(3):723-31.  [Content Brief]

  . Young-Gqamana B, et al. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients. PLoS One. 2013;8(3):e57631.  [Content Brief]

  [4]. Welford RWD, et al. Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. Hum Mol Genet. 2018 Oct. 27(19):3392-3403.  [Content Brief]