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Cystic Fibrosis Therapy Comes of Age, 21 Years After CFTR Discovery

Anil Mehta, MBBS, MSc, FRCP, FRCPCH

Cystic fibrosis (CF) has reached a tipping point in its evolutionary journey from universally fatal outcomes to the possibility of targeted, restorative treatment through novel therapies. The most dramatic illustration of this sea change in fortunes was highlighted recently in a paper published in the New England Journal of Medicine, which reported that the clinically relevant outcome of the universally recognized gold standard for a non-genetic diagnosis of CF, namely a sweat test showing an elevated level of chloride, was partially reversed by a new drug, VX-770 [1].

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