Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith

摘要

Provided is the use of the compound of formula (I) as depicted, with defined substituents, in the preparation of a medicament for use in a subject for the treatment of duchenne muscular dystrophy, wherein the subject has a nonsense mutation in the codon corresponding to position 1417, 3625, or 492 of the dystrophin protein.