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1. (WO2015042685) INHIBITORS OF POLYNUCLEOTIDE REPEAT-ASSOCIATED RNA FOCI AND USES THEREOF

Pub. No.:    WO/2015/042685    International Application No.:    PCT/CA2013/050730
Publication Date: Fri Apr 03 01:59:59 CEST 2015 International Filing Date: Thu Sep 26 01:59:59 CEST 2013
IPC: C07D 231/14
A61K 31/18
A61K 31/352
A61K 31/36
A61K 31/4155
A61K 31/4406
A61K 31/4453
A61K 31/496
A61K 31/5375
A61P 21/00
C07C 311/46
C07D 213/82
C07D 217/08
C07D 265/30
C07D 295/26
C07D 311/84
C07D 317/68
C07D 409/12
G01N 33/50
Applicants: VALORISATION-RECHERCHE, LIMITED PARTNERSHIP
Inventors: CHARTRAND, Pascal
QUERIDO, Emmanuelle
Title: INHIBITORS OF POLYNUCLEOTIDE REPEAT-ASSOCIATED RNA FOCI AND USES THEREOF
Abstract:
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.