Migalastat-d5

Migalastat-d5 (GR181413A-d5 (free base) ) is deuterium labeled Migalastat. Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC50 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].

GR181413A-d5 (free base)

12352005

Glycosidase; Isotope-Labeled Compounds

Metabolic Enzyme/Protease; Others

Metabolism-protein/nucleotide metabolism

Others

O[C@]1(CN[C@@H]([C@](O)([C@]1([2H])O)[2H])C(O)([2H])[2H])[2H]

C6H8D5NO4

168.20

[1]Russak EM, et al. Impact of Deuterium Substitution on the Pharmacokinetics of Pharmaceuticals. Ann Pharmacother. 2019;53 (2) :211-216.|[2]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.|[3]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.|[4]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.|[5]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.

Room temperature

http://file.medchemexpress.com/batch_PDF/HY-W728096/

Isotope-Labeled Compounds

No Development Reported