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Migalastat (hydrochloride)

Migalastat (hydrochloride)

Item no.	HY-14929A-25mg
Manufacturer	MedChem Express
CASRN	75172-81-5
Amount	25 mg
Quantity options	100 mg 10 mM/1 mL 10 mg 1 mg 250 mg 25 mg 50 mg 5 mg
Category	Inhibitors & Compound Libraries Small Molecules
Type	Inhibitors
Specific against	other
Purity	99.0
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.
Smiles	O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O.Cl
ECLASS 10.1	32160490
ECLASS 11.0	32160490
UNSPSC	12000000
Alias	GR181413A
Shipping Condition	Room temperature
Available
Manufacturer - Type	Reference compound
Manufacturer - Targets	Glycosidase
Shipping Temperature	Room Temperature
Storage Conditions	4°C (Powder, sealed storage, away from moisture)
Molecular Weight	199.63
Product Description	Migalastat (GR181413A free base) hydrochloride 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].
Manufacturer - Research Area	Others
Solubility	H2O: ≥ 200 mg/mL
Manufacturer - Pathway	Metabolic Enzyme/Protease
Clinical information	Launched

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HY-14929A-25mg-safety-datasheet.pdf