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Duchenne muscular dystrophy treatment
专利权人:
SUMMIT CORPORATION PLC
发明人:
WYNNE, GRAHAM MICHAEL,WREN, STEPHEN PAUL,JOHNSON, PETER DAVID,PRICE, PAUL DAMIEN,DE MOOR, OLIVIER,NUGENT, GARY,STORER, RICHARD,PYE, RICHARD JOSEPH,DORGAN, COLIN RICHARD
申请号:
ES07705368
公开号:
ES2522290T3
申请日:
2007.02.09
申请国别(地区):
ES
年份:
2014
代理人:
摘要:
Compound of formula (1), or a pharmaceutically acceptable salt thereof, for use in the treatment or prophylaxis of Duchenne muscular dystrophy or Becker muscular dystrophy, wherein X is O; Y is N; R9 is -L-R3; L is (i) a simple link; or (ii) a linker group selected from: (a) -O-, -S- and - (CO) nNR18-; (b) alkylene, alkenylene and alkynylene, each of which is optionally interrupted by one or more of -O-, -S- or -NR18-, or one or more single, double or triple C-C bond; and (c) a single or double -N-N-bond; in which n is an integer from 0 to 2; and R18 is hydrogen, alkyl or C (O) R16; R3 is alkyl, alkoxy or aryl, each of which is optionally substituted with one to three R2, which may be the same or different; each occurrence of R2 is independently alkyl optionally substituted with one or more of halogen, alkoxy, optionally substituted aryl, optionally substituted thioaryl or optionally substituted aryloxy; optionally substituted alkoxy with optionally substituted alkyl or aryl; hydroxyl; optionally substituted aryl; thioalkyl optionally substituted with optionally substituted alkyl or aryl; thioaryl in which the aryl is optionally substituted; halogen; NO2; CN; OC (>; = W) NR10R11; NR10R11; SO2R12; NR13SO2R14; C (>; = W) R16; NR15C (>; = W) R17; or P (>; = O) OR40R41; W is O, S or NR20; A1, A2, A3 and A4, which may be the same or different, are N or CR1, in which all of A1-A4 are CR1, or one of A1-A4 is nitrogen; each occurrence of R1 is independently (i) hydrogen; or (ii) R2; wherein at least one occurrence of R1 is NR15C (>; = W) R17; NHCONHR15 ', wherein R15' is phenyl, C1-C6 alkyl or CO-phenyl optionally substituted with one or more of halogen; NHSO2- (C1-C6 alkyl); NHSO2-phenyl; SO2- (C1-C6 alkyl); or P (>; = O) OR40R41; when in an adjacent pair of A1-A4 each represents CR1, adjacent carbon atoms together with their substituents R1 optionally form a ring B; R10 and R11 are each independently hydrogen, optionally substituted alkyl with optionally subst
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