1-oxo-isoindoline compounds for the treatment of spinal...

C - Chemistry – Metallurgy – 07 – D

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C07D 209/46 (2006.01) A61K 31/4035 (2006.01) A61P 25/16 (2006.01)

Patent

CA 2645426

Compounds of Formula (I) or (Il) useful for the treatment of spinal muscular atrophy or other uses, 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 by mutation or frameshift.

La présente invention concerne des composés pouvant être employés dans le traitement de l'amyotrophie spinale ainsi que dans d'autres applications, de même que des méthodes d'utilisation de tels composés pour augmenter l'expression de SMN, augmenter l'expression de EAAT2 ou augmenter l'expression d'un acide nucléique qui code pour un codon stop traductionnel introduit par mutation ou déphasage.

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