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Migalastat hydrochloride

CAS No. : 75172-81-5

MCE 国际站：Migalastat hydrochloride

产品活性：Migalastat hydrochloride (GR181413A) 是一种有效和竞争性的 α-galactosidase A 抑制剂，对人 α-Gal A 的 IC₅₀ 值为 0.04 μM。

研究领域：Others

作用靶点：Others

In Vitro: Both IC₅₀ and K_i values of Migalastat hydrochloride (GR181413A) 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.
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM).
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment.
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively.

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