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    Treatment of Duchenne muscular dystrophy
    3.
    发明授权
    Treatment of Duchenne muscular dystrophy 有权
    杜氏肌营养不良症的治疗

    公开(公告)号：US08518980B2

    公开(公告)日：2013-08-27

    申请号：US12278769

    申请日：2007-02-09

    申请人： Graham Michael Wynne ,  Stephen Paul Wren ,  Peter David Johnson ,  Paul Damien Price ,  Olivier De Moor ,  Gary Nugent ,  Richard Storer ,  Richard Joseph Pye ,  Colin Richard Dorgan

    发明人： Graham Michael Wynne ,  Stephen Paul Wren ,  Peter David Johnson ,  Paul Damien Price ,  Olivier De Moor ,  Gary Nugent ,  Richard Storer ,  Richard Joseph Pye ,  Colin Richard Dorgan

    IPC分类号： A61K31/42 ,  A01N43/76 ,  C07D263/57

    CPC分类号： C07F9/4003 ,  A61K31/343 ,  A61K31/404 ,  A61K31/4184 ,  A61K31/4192 ,  A61K31/4196 ,  A61K31/423 ,  A61K31/4245 ,  A61K31/426 ,  A61K31/4355 ,  A61K31/4365 ,  A61K31/437 ,  A61K31/4439 ,  A61K31/4525 ,  A61K31/454 ,  A61K31/4709 ,  A61K31/496 ,  A61K31/506 ,  A61K31/517 ,  A61K31/5377

    摘要： There are disclosed compound of Formula (1): A1, A2, A3 and A4 which may be the same or different, represent N or CR1, X is a divalent group selected from O, S(O)n, C═W, NR4, NC(═O)R5 and CR6R7, W is O, S, NR20, Y is N or CR8, one of R4, R5, R6, R8, R9 and NR20 represents -L-R3, in which L is a single bond or a linker group, additionally, R1, R3-R9, which may be the same or different, independently represent hydrogen or a substituent and R20 represents hydrogen, hydroxyl, alkyl optionally substituted by aryl, alkoxy optionally substituted by aryl, aryl, CN, optionally substituted alkoxy, optionally substituted aryloxy, optionally substitute alkanoyl, optionally substituted aroyl, NO2, NR30R31, in which R30 and R31, which may be the same or different, represent hydrogen, optionally substituted alkyl or optionally substituted aryl; additionally, one of R30 and R31 may represent optionally substituted alkanoyl or optionally substituted aroyl, n represents an integer from 0 to 2, in addition, when an adjacent pair of A1-A4 each represent CR1, then the adjacent carbon atoms, together with their substituents may form a ring B, when X is CR6R7, R6 and R7, together with the carbon atom to which they are attached may form a ring C, or a pharmaceutically acceptable salt thereof, in the manufacture of a medicament for the therapeutic and/or prophylactic treatment of Duchenne muscular dystrophy, Becker muscular dystrophy or cachexia.

    摘要翻译： 公开了式（1）的化合物：A1，A2，A3和A4可以相同或不同，表示N或CR1，X是选自O，S（O）n，C = W，NR4的二价基团 ，NC（= O）R5和CR6R7，W是O，S，NR20，Y是N或CR8，R4，R5，R6，R8，R9和NR20之一表示-L-R3，其中L是单键 或连接基团，另外，可以相同或不同的R 1，R 3 -R 9独立地表示氢或取代基，R 20表示氢，羟基，任选被芳基取代的烷基，任选被芳基，芳基，CN， 任选取代的烷氧基，任选取代的芳氧基，任选取代的烷酰基，任选取代的芳酰基，NO 2，NR 30 R 31，其中R 30和R 31可以相同或不同，表示氢，任选取代的烷基或任选取代的芳基; 此外，R30和R31中的一个可以表示任选取代的烷酰基或任选取代的芳酰基，n表示0至2的整数，此外，当相邻的一对A1-A4各自表示CR1时，则相邻的碳原子连同它们 取代基可以形成环B，当X是CR 6 R 7，R 6和R 7时，连同它们所连接的碳原子一起形成环C或其药学上可接受的盐在制备用于治疗和/ 或预防性治疗杜氏肌营养不良症，贝克肌营养不良症或恶病质。