TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

摘要

There are discloses compounds of formula (I): wherein A¹, A², A³ and A⁴, which may be the same or different, represent N or CR¹ X, Y and Z, which may be the same or different, represent O, S(O)_n, C=W, NR⁴, NC(=O)R⁵ and CR⁶R⁷, W iS O, S, NR²⁰, one of R⁴, R⁵, R⁶, and R²⁰ represents - L -R³, in which L is a single bond or a linker group, additionally, R³, R⁴, R ⁵, R⁶, and R⁷, which may be the same or different, independently represent hydrogen or a substituent and R₂₀ represents hydrogen, aryl, hydroxyl, alkyl optionally substituted by aryl, alkoxy optionally substituted by aryl, CN, optionally substituted alkoxy, optionally substituted aryloxy, optionally substituted alkanoyl, optionally substituted aroyl, 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, an adjacent pair of X, Y and Z cannot both represent O, or S or together represent O - S, any pair of R⁶, R⁷ and R⁴ may when attached to adjacent atoms together represent a single bond between the adjacent atoms to which they are attached, 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, Y or Z 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.