ALMSTEAD, NEIL G,CHEN, GUANGMING,HIRAWAT, SAMIT,HWANG, SEONGWOO,KARP, GARY M,KENNEDY, PAUL,MILLER, LANGDON,MOON, YOUNG-CHOON,REN, HONGYU,TAKASUGI, JAMES J,WELCH, ELLEN M,WILDE, RICHARD G
申请号：
NZ59696507
公开号：
NZ596965A
申请日:
2007.03.29
申请国别(地区):
NZ
年份:
2013
代理人:
摘要：
596965 Disclosed is the use of a compound having the formula defined herein, or a pharmaceutically acceptable salt, solvate or hydrate thereof, in the treatment of cystic fibrosis, wherein the subject has a nonsense mutation in the codon corresponding to position 414, 493, 1316, 553, 542, 1162,122, 1455, 822, 60, 764, 1291, 849, 434, 88, 1158 or 6542 of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Specific embodiments of the compound include: 3-[S-2-fluoro-phenyl-[1,2,4]oxadiazol-3-yl]-benzoic acid (PTC124).
