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| Ataluren Publications |
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Eitan Kerem et al. (2008). Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial. The Lancet, 372:719-27. |
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Ming Du, Xiaoli Liu, Ellen M. Welch, Samit Hirawat, Stuart W. Peltz, and David M. Bedwell. (2008). PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model. Proceedings of the National Academy of Sciences, 105(6):2064-2069. |
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Ellen M. Welch et al. (2007). PTC124 targets genetic disorders caused by nonsense mutations. Nature, 447:87-91. |
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Samit Hirawat et al. (2007). Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-Dose Administration to Healthy Male and Female Adult Volunteers. Journal of Clinical Pharmacology, 47(4):430-444. |
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