[EN] INHIBITORS OF POLYNUCLEOTIDE REPEAT-ASSOCIATED RNA FOCI AND USES THEREOF [FR] INHIBITEURS D'AGRÉGATS D'ARN ASSOCIÉS À LA RÉPÉTITION DE POLYNUCLÉOTIDES ET LEURS UTILISATIONS
[EN] INHIBITORS OF POLYNUCLEOTIDE REPEAT-ASSOCIATED RNA FOCI AND USES THEREOF<br/>[FR] INHIBITEURS D'AGRÉGATS D'ARN ASSOCIÉS À LA RÉPÉTITION DE POLYNUCLÉOTIDES ET LEURS UTILISATIONS
申请人:VALORISATION RECH
公开号:WO2015042685A1
公开(公告)日:2015-04-02
Compounds which inhibit the formation and/or accumulation of RNA foci, such as those due to polynucleotide repeats (e.g., trinucleotide repeats) are described herein. Also described herein are uses of such compounds, such as for the inhibition of the formation and accumulation such RNA foci, as well as for the treatment of polynucleotide repeat disorders (e.g., trinucleotide repeat disorders), such as myotonic dystrophy (e.g., DM1). Such compounds include compounds of formula 1, 1a, 1b, 2, 2a and (3) described herein.