Description
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.
Product information
CAS Number: 75172-81-5
Molecular Weight: 199.63
Formula: C6H14ClNO4
Chemical Name: (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride
Smiles: Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
InChiKey: ZJIHMALTJRDNQI-OLALXQGDSA-N
InChi: InChI=1S/C6H13NO4.ClH/c8-2-3-5(10)6(11)4(9)1-7-3;/h3-11H,1-2H2;1H/t3-,4+,5+,6-;/m1./s1
Technical Data
Appearance: Solid Power
Purity: ≥98% (or refer to the Certificate of Analysis)
Solubility: H2O : ≥ 200 mg/mL (1001.85 mM).
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical or refer to Certificate of Analysis
Storage Condition: Dry, dark and -20 oC for 1 year or refer to the Certificate of Analysis.
Shelf Life: ≥12 months if stored properly.
Stock Solution Storage: 0 - 4 oC for 1 month or refer to the Certificate of Analysis.
Drug Formulation: To be determined
HS Tariff Code: 382200
How to use
In Vitro:
Both IC50 and Ki values of Migalastat (GR181413A) hydrochloride toward human lysosomal a-Gal A are 0.04 μM.
In Vivo:
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.
Products are for research use only. Not for human use.
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