Compounds for nonsense suppression and methods for their use
View Patent ↗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.
1. A method of treating Duchenne muscular dystrophy associated with a nonsense mutation in the dystrophin gene in a patient in need thereof, wherein the nonsense mutation results in a premature termination codon in mRNA, wherein the premature termination codon results in either or both premature mRNA translation termination or nonsense-mediated mRNA decay, comprising administering to the patient an effective amount of a compound selected from the group consisting of:
and a pharmaceutically acceptable salt, racemate or stereoisomer thereof.