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Migalastat

CAT: 0804-HY-14929Size: 1 EachDry Ice: NoHazardous: No
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CAT#:0804-HY-14929Size:1 Each
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Description
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].
CAS Number
108147-54-2
Product Name Alternative
GR181413A (free base) ; 1-Deoxygalactonojirimycin
UNSPSC
12352005
Hazard Statement
H315-H319-H320
Target
Glycosidase
Type
Reference compound
Related Pathways
Metabolic Enzyme/Protease
Field of Research
Others
Assay Protocol
https://www.medchemexpress.com/migalastat.html
Solubility
10 mM in DMSO
Smiles
O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O
Molecular Formula
C6H13NO4
Molecular Weight
163.17
Precautions
P264-P280-P302+P352-P305+P351+P338-P362
References & Citations
[1]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.|[2]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.|[3]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.|[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.
Shipping Conditions
Room temperature
Scientific Category
Reference compound1
Clinical Information
Launched

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