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VX-809(Lumacaftor)

编号: 92219
Cas号: 936727-05-8
纯度: 98% 

From Wikipedia, the free encyclopedia

Lumacaftor (USAN, codenamed VX-809) is an experimental drug for the treatment of cystic fibrosis being developed by Vertex Pharmaceuticals. The drug is designed to be effective in patients that have the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), the defective protein that causes the disease. F508del, meaning that the amino acid phenylalanine in position 508 is missing, is found in about 60% of cystic fibrosis patients in Europe, and in about 90% of persons with some mutation in the CFTR gene.

Lumacaftor acts on F508 CFTR during protein folding and suppresses misfolding.

Results from a Phase II clinical trial indicate that patients with the most common form of genetic mutation causing cystic fibrosishomozygous F508delhad a mean increase of 7.4% in lung function (FEV1) on a combination of lumacaftor and ivacaftor.However these results did not show a significant improvement in lung function.

 

仅供研究使用。 我们不向患者出售。

化学信息

名称VX-809(Lumacaftor)
Iupac 化学名称3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid 
同义词VX809; VX 809 
英文同义词VX809; VX 809 
分子式C24H18F2N2O5 
分子量452.41 
SmileO=C(O)C1=CC=CC(C2=NC(NC(C3(C4=CC=C(OC(F)(F)O5)C5=C4)CC3)=O)=CC=C2C)=C1
InChiKeyUFSKUSARDNFIRC-UHFFFAOYSA-N
InChiInChI=1S/C24H18F2N2O5/c1-13-5-8-19(27-20(13)14-3-2-4-15(11-14)21(29)30)28-22(31)23(9-10-23)16-6-7-17-18(12-16)33-24(25,26)32-17/h2-8,11-12H,9-10H2,1H3,(H,29,30)(H,27,28,31)
Cas号936727-05-8
相关CAS号

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外观性状Solid powder 
纯度98% 
存储-20 ºC for 3 years 
可溶性Soluble in DMSO 
处理方式
运输条件Shipped under ambient temperature 
海关编码
Coming soon.
Targets
Mechanism
Cell study
Animal study
Clinical study
Not available

化学结构

92219 - VX-809(Lumacaftor) | CAS 936727-05-8

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