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Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

13 Jan 2011

Abstract excerpt

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 ataluren three times daily (morning, midday and evening) for 12...

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PMID: 21233271
DOI: 10.1183/09031936.00120910

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