发明申请
- 专利标题: Compositions and methods for treating amyloidosis
- 专利标题(中): 治疗淀粉样变性的组合物和方法
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申请号: US13066451申请日: 2011-04-14
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公开(公告)号: US20110281951A1公开(公告)日: 2011-11-17
- 发明人: Walter Szarek , Donald Weaver , Xianqi Kong , Heather Gordon
- 申请人: Walter Szarek , Donald Weaver , Xianqi Kong , Heather Gordon
- 主分类号: A61K31/185
- IPC分类号: A61K31/185 ; A61P3/10 ; A61P25/00 ; A61P25/28
摘要:
Therapeutic compounds and methods for modulating amyloid aggregation in a subject, whatever its clinical setting, are described. Amyloid aggregation is modulated by the administration to a subject of an effective amount of a therapeutic compound of the formula or a pharmaceutically acceptable salt or ester, such that modulation of amyloid aggregation occurs. R1 and R2 are each independently a hydrogen atom or a substituted or unsubstituted aliphatic or aryl group. Z and Q are each independently a carbonyl (C═O), thiocarbonyl (C═S), sulfonyl (SO2), or sulfoxide (S═O) group. “k” and “m” are 0 or 1, provided when k is 1, R1 is not a hydrogen atom, and when m is 1, R2 is not a hydrogen atom. In an embodiment, at least one of k or m must equal 1. “p” and “s” are each independently positive integers selected such that the biodistribution of the therapeutic compound for an intended target site is not prevented while maintaining activity of the therapeutic compound. T is a linking group and Y is a group of the formula -A X wherein A is an anionic group at physiological pH, and X is a cationic group.
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