Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.


In a subset of patients with cystic fibrosis (CF), nonsense mutations (premature stop codons) disrupt production of full-length, functional CF transmembrane conductance regulator (CFTR). Ataluren (PTC124) allows ribosomal readthrough of premature stop codons in mRNA. We evaluated drug activity and safety in patients with nonsense mutation CF who took… (More)
DOI: 10.1183/09031936.00120910


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@article{Wilschanski2011ChronicA, title={Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis.}, author={Michael Wilschanski and Langdon L. Miller and David Shoseyov and Hannah Blau and Joseph Rivlin and Micha Aviram and Morton L. Cohen and Shoshana Armoni and Yasmin Yaakov and Thea Pugatsch and Malena Cohen-Cymberknoh and Nilsen L Miller and Ali Reha and Valerie J. Northcutt and Samit Hirawat and Katelyn Donnelly and Gary L. Elfring and Temitayo Ajayi and Eitan Kerem}, journal={The European respiratory journal}, year={2011}, volume={38 1}, pages={59-69} }