Compounds for nonsense suppression, and methods for their use

C - Chemistry – Metallurgy – 07 – D

Patent

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Details

C07D 271/06 (2006.01) A61K 31/4245 (2006.01) A61P 25/28 (2006.01) A61P 35/00 (2006.01) C07D 413/12 (2006.01)

Patent

CA 2583177

The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.

La présente invention a pour objet des méthodes, des composés et des préparations permettant le traitement prophylactique ou thérapeutique de maladies liées à la présence de mutations non-sens dans un mARN, en administrant les composés ou compositions décrits dans la présente invention. La présente invention décrit plus particulièrement des méthodes, des composés et des préparations permettant de supprimer les terminaisons prématurées de traduction liées à la présence de mutations non-sens dans un mARN.

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