Ataluren

Description: Ataluren (PTC124) is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. Target: CFTR, Autophagy. Smiles: FC1=C(C2=NC(=NO2)C3=CC(C(O)=O)=CC=C3)C=CC=C1. References: Welch EM, et al. Nature, 2007, 447(7140), 87-91.