TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

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.