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75172-81-5

中文名稱 脫氧半乳糖野生霉素 鹽酸鹽
英文名稱 DEOXYGALACTONOJIRIMYCIN, HYDROCHLORIDE
CAS 75172-81-5
分子式 C6H14ClNO4
分子量 199.63
MOL 文件 75172-81-5.mol
75172-81-5 結(jié)構(gòu)式 75172-81-5 結(jié)構(gòu)式

基本信息

中文別名
脫氧半乳糖苷酶抑素鹽酸鹽
脫氧半乳糖野生霉素 鹽酸鹽
英文別名
DGJ
Amigal
Migalastat HCl
Unii-cly7m0xd20
GALACTOSTATIN HCL
DGJ, HYDROCHLORIDE
Migalastat hydrochloride
Galactostatin hydrochloride
DEOXYGALACTONOJIRIMYCIN HCL
1-DEOXYGALACTONOJIRIMYCIN HCL
所屬類別
有機(jī)原料:羧酸類化合物及衍生物

物理化學(xué)性質(zhì)

熔點(diǎn)260
儲(chǔ)存條件2-8°C
溶解度少許溶于甲醇和水
形態(tài)白色結(jié)晶固體
顏色白色至棕色

安全數(shù)據(jù)

危險(xiǎn)性符號(hào)(GHS)GHS hazard pictograms
GHS07
警示詞警告
危險(xiǎn)性描述H302-H315-H319-H335
危險(xiǎn)品標(biāo)志Xi
危險(xiǎn)類別碼36/37/38
安全說(shuō)明26-36
WGK Germany3
Hazard NoteIrritant
海關(guān)編碼29333990

常見(jiàn)問(wèn)題列表

用途

脫氧半乳糖野生霉素鹽酸鹽是一種有效和競(jìng)爭(zhēng)性的α-galactosidase A抑制劑,對(duì)人α-Gal A的 IC50值為0.04 μM。

靶點(diǎn)

IC50: 0.04 μM (human α-Gal A); Ki: 0.04 μM (human α-Gal A)

體外研究

Both IC 50 and K i values of Migalastat (GR181413A) hydrochloride toward human lysosomal a-Gal A are 0.04 μM.

體內(nèi)研究

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.

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