Isoindoline compounds for the treatment of spinal muscular...

C - Chemistry – Metallurgy – 07 – D

Patent

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C07D 209/46 (2006.01) A61K 31/395 (2006.01) A61K 31/4035 (2006.01) A61K 31/404 (2006.01) A61P 25/00 (2006.01) A61P 31/00 (2006.01) C07D 401/04 (2006.01) C07D 403/04 (2006.01) C07D 405/04 (2006.01) C07D 409/04 (2006.01) C07D 413/04 (2006.01) C07D 417/04 (2006.01) C07D 471/04 (2006.01) C07D 487/04 (2006.01) C07D 491/056 (2006.01)

Patent

CA 2700841

Disclosed is a compound of Formula (I) in which W and R1-R6 are defined herein. Also disclosed is a method of treating spinal muscular atrophy, as well as methods of using such compounds to increase SMN expression, increase EAAT2 expression, or increase the expression of a nucleic acid that encodes a translational stop codon introduced directly or indirectly by mutation or frameshift.

La présente invention concerne un composé de formule (I), W et R1 à R6 étant définis ici. L'invention concerne également un procédé de traitement d'une amyotrophie spinale, ainsi que des procédés d'utilisation de tels composés pour augmenter l'expression de la SMN, augmenter l'expression de l'EAAT2, ou augmenter l'expression d'un acide nucléique qui code pour un codon d'arrêt traductionnel introduit directement ou indirectement par mutation ou déphasage.

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