首页> 外国专利> Hydroxy piperidine derivatives, a method for the synthesis of said derivatives, an in vitro method for inhibiting, enhancing the activity and stabilizing glucocerebrosidase, a pharmaceutical composition, an in vitro method for inhibiting a beta-glucosidase and use of said derivatives in the preparation of a medicament

Hydroxy piperidine derivatives, a method for the synthesis of said derivatives, an in vitro method for inhibiting, enhancing the activity and stabilizing glucocerebrosidase, a pharmaceutical composition, an in vitro method for inhibiting a beta-glucosidase and use of said derivatives in the preparation of a medicament

机译：羟基哌啶衍生物，所述衍生物的合成方法，抑制，增强活性和稳定葡糖脑苷脂酶的体外方法，药物组合物，抑制β-葡糖苷酶的体外方法以及所述衍生物在制备β-葡萄糖苷中的用途药物

页面导航

摘要
著录项
相似文献

摘要

The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as active-site specific chaperones for the mutant glucocerebrosidase associated with the disease.

机译：本发明提供了新颖的羟基哌啶（HP）衍生物，其（i）在对应于吡喃糖环的异头位置的位置具有正电荷; （ii）从环氧在吡喃糖中的相应位置发出的短而柔性的连接子; （iii）与连接体连接的亲脂部分及其药学上可接受的盐。如果亲脂性部分对应于具有6个或更多个碳的线性长度的烃链，则可以不存在该连接基。本发明还提供了一种通过将新的HP衍生物作为与疾病相关的突变型葡糖脑苷脂酶的活性部位特异性伴侣而给药的方法来治疗患有高雪氏病的个体。

著录项

公开/公告号IL175444A

专利类型
公开/公告日2013-05-30

原文格式PDF
申请/专利权人 AMICUS THERAPEUTICS INC.;
展开▼

申请/专利号IL20060175444
发明设计人
展开▼

申请日2006-05-04
分类号A61K;A61K31/437;A61K31/445;A61K31/454;A61K31/4745;A61K31/50;A61K31/501;A61K48;A61P;A61P43;C07D;C07D211/40;C07D471/02;C07D471/04;
国家 IL
入库时间 2022-08-21 16:41:36

相似文献

专利
外文文献
中文文献