[EN] COMPOSITIONS FOR PROMOTING READTHROUGH OF PREMATURE TERMINATION CODONS, AND METHODS OF USING THE SAME [FR] COMPOSITIONS PERMETTANT DE FAVORISER LA TRANSLECTURE DE CODONS DE TERMINAISON PRÉMATURÉE, ET LEURS PROCÉDÉS D'UTILISATION
[EN] COMPOSITIONS FOR PROMOTING READTHROUGH OF PREMATURE TERMINATION CODONS, AND METHODS OF USING THE SAME [FR] COMPOSITIONS PERMETTANT DE FAVORISER LA TRANSLECTURE DE CODONS DE TERMINAISON PRÉMATURÉE, ET LEURS PROCÉDÉS D'UTILISATION
[EN] COMPOSITIONS FOR PROMOTING READTHROUGH OF PREMATURE TERMINATION CODONS, AND METHODS OF USING THE SAME<br/>[FR] COMPOSITIONS PERMETTANT DE FAVORISER LA TRANSLECTURE DE CODONS DE TERMINAISON PRÉMATURÉE, ET LEURS PROCÉDÉS D'UTILISATION
申请人:THE CENTRE FOR DRUG RES AND DEV
公开号:WO2017049409A1
公开(公告)日:2017-03-30
Disclosed are compounds of general formula (I) that promote readthrough of a premature termination codon (PTC) of an RNA molecule in a translation system, and their use, alone or in combination with other compounds, such as aminoglycoside, to treat diseases or disorders ameliorated by modulation of a premature termination codon (PTC) of an RNA molecule in a translation system. The disorder or disease may be Dystrophic epidermolysis bullosa, Batten disease, Duchenne muscular dystrophy, cancer, and spinal muscular atrophy. Ar-L-B (I)