- 英文名称
- Migalastat hydrochloride
- 分子式
- C6H14ClNO4
- 分子量
- 199.63
- 中文别名
- 脱氧半乳糖野生霉素 盐酸盐; 1-脱氧半乳糖野尻霉素盐酸盐; 脱氧半乳糖诺吉霉素; 1-脱氧半乳糖诺吉霉素盐酸盐; (2R,3S,4R,5S)-2-甲醇哌啶-3,4,5-三醇盐酸盐; (2R,3S,4R,5S)-2-(Hydroxymethyl)piperidine-3,4,5-triol hydrochloride; deoxygalactonojirimycin, hydrochloride; Deoxygalactonojirimycin Hydrochloride; (1R,3R,4R,5S)-3,4,5-trihydroxy-2-(hydroxymethyl)piperidinium chloride; 1,5-dideoxy-1,5-imino-D-glucitol hydrochloride; 1-Deoxymannojirimycinehydrochloride; DEOXYMANNONOJIRIMYCIN HCL; (+)-(2R,3R,4R,5S)-2-hydroxymethyl-3,4,5-trihydroxypiperidine hydrochloride; DEOXYMANNOJIRIMYCIN,HYDROCHLORIDE; (+)-1-deoxynojirimycin hydrochloride; 1-DEOXYMANNOJIRIMYCIN HCL; DMM; DMJ,HYDROCHLORIDE; Migalastat (hydrochloride); 1,5-DIDEOXY-1,5-IMINO-D-GALACTITOL, HYDROCHLORIDE; 1-DEOXYGALACTONOJIRIMYCIN HCL; DGJ; DGJ, HYDROCHLORIDE; GALACTOSTATIN HCL; 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride, (2R,3S,4R,5S)-
- 用途
- 作为有效和竞争性的α-galactosidase A抑制剂,对人α-Gal A的IC50值为0.04 μM;在转基因小鼠中,经其处理后,心脏、肾脏、脾脏和肝脏中的α-Gal A活性呈剂量和时间依赖性增加;口服给药可降低法布里转基因小鼠的组织GL-3水平,以及部分法布里病患者尿液和肾脏中的GL-3水平;还可降低转基因小鼠肾脏、心脏和皮肤中升高的lyso-Gb3水平,分别高达64%、59%和81%