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Pack Size | Price | Availability | Quantity |
---|---|---|---|
1 mg | $33 | In Stock | |
2 mg | $44 | In Stock |
Description | Migalastat hydrochloride (GR181413A) is an orally available, potent and competitive inhibitor of alpha-galactosidase A. It promotes the transport of alpha-galactosidase A to the lysosome and can be used in the study of Fabry disease. |
In vitro | Migalastat hydrochloride (GR181413A) demonstrates IC50 and Ki values of 0.04 μM for human lysosomal alpha-Gal A[4]. |
In vivo | Fabry disease is an X-linked recessive disorder caused by deficient activity of alpha-galactosidase A (α-Gal A)[2]. In transgenic mice expressing the mutant human α-Gal A (TgM), oral administration of Migalastat hydrochloride (3 mg/kg per day for 4 consecutive weeks) results in a dose- and time-dependent increase in α-Gal A activity in the heart, kidneys, spleen, and liver[2].After a 2-week pretreatment with Migalastat hydrochloride, the half-life for all major issues is less than 1 day[2]. Administration of Migalastat hydrochloride (100 mg/kg per day orally for 28 days) in transgenic mice leads to a reduction of 64%, 59%, and 81% in globotriaosylceramide (Gb3) levels in the kidneys, heart, and skin, respectively[3]. |
Alias | GR181413A hydrochloride, Migalastat HCl, GR181413A HCl |
Molecular Weight | 199.63 |
Formula | C6H14ClNO4 |
Cas No. | 75172-81-5 |
Storage | store at low temperature Powder: -20°C for 3 years | In solvent: -80°C for 1 year | |||||||||||||||||||||||||||||||||||
Solubility Information | H2O: 50 mg/mL (250.46 mM) | |||||||||||||||||||||||||||||||||||
Solution Preparation Table | ||||||||||||||||||||||||||||||||||||
H2O
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