Compositions, and kits comprising targeted aldehyde or acetal protease inhibitor compounds for treating muscle disorders
申请人:Stracher Alfred
公开号:US20080039531A1
公开(公告)日:2008-02-14
Described herein are compounds that comprise a carrier residue that is or is an analog of carnitine, an optional linker group, and a residue of a protease inhibitor comprising an aldehyde group or acetal derivatives of the aldehyde group, and pharmaceutical compositions and kits thereof. The compounds, compositions, and kits are useful for treating muscle disorders in animals and humans that result from the undesired activity of biological proteases. The compounds of the invention are particularly effective as calpain inhibitors, and can be used to treat the resulting muscular disorders, exemplified by Duchenne or Becker muscular dystrophy.
本文介绍的是一种化合物,该化合物包括一个载体残基,该载体残基是肉碱或其类似物,一个可选的连接基团,以及一个蛋白酶抑制剂残基,该蛋白酶抑制剂残基包括一个醛基或醛基的缩醛衍生物,以及其制备的药物组合物和试剂盒。这些化合物、组合物和试剂盒可用于治疗动物和人类的肌肉疾病,这些肌肉疾病是由生物蛋白酶的不良活性引起的。本发明的化合物特别有效地作为卡尔帕因抑制剂,并可用于治疗由此引起的肌肉疾病,例如杜氏或贝克肌肉萎缩症。