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Cystic fibrosis: a novel therapeutic angle or a false dawn
  1. P M George
  1. Dr P M George, ST2, Respiratory Medicine, Royal Brompton Hospital, London, UK; petergeorge{at}

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Worldwide, approximately 10% of patients with cystic fibrosis have premature stop codons in the mRNA for the CFTR gene. These result in phenotypically severe variants of cystic fibrosis. PTC124 is a small molecule available as an oral preparation which allows the ribosome to selectively ignore these mutations and hence produce the functioning protein.

This prospective phase II trial recruited …

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