Migalastat HCl, also known as AT1001 or GR181413A, is a pharmacological chaperone that selectively binds, stabilizes, and increases cellular levels of -Gal A. Oral administration of migalastat HCl reduces tissue GL-3 in Fabry transgenic mice, and in urine and kidneys of some FD patients. Migalastat HCl may provide a potential novel genotype-specific treatment for Fabry Disease (FD). Phase 3 studies are ongoing. Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme -galactosidase A (-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues.
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化学信息
名称 | Migalastat HCl |
Iupac 化学名称 | Migalastat HCl |
同义词 | AT1001; AT 1001; AT-1001; GR181413A; GR 181413A; GR-181413A; 1,5-Dideoxy-1,5-imino-D-galactitol; 1-Deoxygalactonojirimycin; 1-Deoxygalactostatin; Amigal; Migalastat |
英文同义词 | AT1001; AT 1001; AT-1001; GR181413A; GR 181413A; GR-181413A; 1,5-Dideoxy-1,5-imino-D-galactitol; 1-Deoxygalactonojirimycin; 1-Deoxygalactostatin; Amigal; Migalastat |
分子式 | C6H13NO4 |
分子量 | 163.17 |
Smile | C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O.Cl |
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 |
Cas号 | 75172-81-5 |
MDL | MFCD00269962 |
相关CAS号 | |
外观性状 | crystalline solid |
纯度 | 98% |
存储 | 3 years -20ºCpowder |
可溶性 | Soluble in DMSO |
处理方式 | |
运输条件 | Shipped under ambient temperature as non-hazardous chemical. |
海关编码 | |
Targets | |
Mechanism | |
Cell study | |
Animal study | |
Clinical study | |