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    • 90. 发明申请
    • COMPOUNDS HAVING ACTIVITY IN CORRECTING MUTANT CFTR CELLULAR PROCESSING
    • 具有修复突变CFTR细胞处理活性的化合物
    • US20120004405A1
    • 2012-01-05
    • US13139243
    • 2009-12-14
    • Peter Prehm
    • Peter Prehm
    • C07H7/02C07C233/54
    • C07C233/81C07C233/25C07H15/203
    • The present invention relates to a compound which is characterized by the formula (I) or a pharmaceutically acceptable salt, solvate, hydrate thereof, wherein the ring systems A and B are independently selected from a monosaccharide, aryl (preferably phenyl), a heteroaryl or cycloalkyl (preferably cyclohexan), preferably with all substituents in equatorial configurations; R1 is independently selected from alkyl (preferably C1 to C6), a substituted or unsubstituted phenyl, preferably CH3; R2 is H, alkyl (preferably C1 to C6), a carbohydrate in a glycosidic β-linkage, preferably H; R3, R4, R5, and R6 are independently selected from H, (OH) hydroxy, alkyl preferably C1 to C6, alkoxy (preferably C1 to C6), amino, alkylamino (preferably C1 to C6), halogen, benzylamino, or benzoylamino; X is O, NH, alkylamino (NR), CO, S; and Y is O, NH, alkylamino (NR), CO, S. The present invention also relates to the compound of the invention and, optionally, a pharmaceutically acceptable carrier, for use in the treatment of (for treating) and/or preventing a disease or medical condition which is associated with mutant CFTR.
    • 本发明涉及以式(I)化合物或其药学上可接受的盐,溶剂合物,水合物为特征的化合物,其中环系统A和B独立地选自单糖,芳基(优选苯基),杂芳基或 环烷基(优选环己烷),优选具有赤道结构中的全部取代基; R 1独立地选自烷基(优选C 1至C 6),取代或未取代的苯基,优选CH 3; R2是H,烷基(优选C1至C6),糖苷键连接的碳水化合物,优选H; R3,R4,R5和R6独立地选自H,(OH)羟基,优选C1至C6烷基,烷氧基(优选C1至C6),氨基,烷基氨基(优选C1至C6),卤素,苄基氨基或苯甲酰氨基; X是O,NH,烷基氨基(NR),CO,S; 并且Y是O,NH,烷基氨基(NR),CO,S。本发明还涉及用于治疗(治疗)和/或预防的本发明化合物和任选的药学上可接受的载体 与突变CFTR相关的疾病或医学病症。