| abstract |
Compound of formula (I) ** Formula ** in which: R1 is hydrogen or an optionally substituted alkyl chain; R2 is hydrogen; an optionally substituted alkyl chain; or an optionally substituted alkenyl; R3 is hydrogen; an optionally substituted alkyl chain; or a linker attached to another compound of formula (I) through N-7, where N-7 is the N atom attached to R3 in the structure of formula (I), wherein the linker is selected from an alkyl optionally substituted, optionally substituted aryl, or optionally substituted alkylarylalkyl; X is O; Cast; Z is hydrogen; an optionally substituted alkyl chain; or OH; or a derivative of said compound of formula (I) represented by the formula: ** Formula ** provided that this compound of formula (I) is not caffeine, for use in reducing the severity of symptoms of type myotonic dystrophy 1 or type 2 in a subject suffering from type 1 or type 2 myotonic dystrophy by increasing the amount of MBNL free protein, such symptoms including those affecting the heart, central nervous system, smooth muscles, hormonal system, system immune, vision, reproductive system and skin. |