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Migalastat HCl

编号: 16123048
Cas号: 75172-81-5
纯度: 98% 

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 
SmileC1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O.Cl
InChiKeyZJIHMALTJRDNQI-OLALXQGDSA-N
InChiInChI=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
MDLMFCD00269962
相关CAS号

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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
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Targets
Mechanism
Cell study
Animal study
Clinical study
Not available

化学结构

16123048 - Migalastat HCl | CAS 75172-81-5

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