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Firefighters, hang up your hoses (not the articles of female attire!)?
These are heady days in the world of cystic fibrosis (CF). Diagnosis is for the most part early by newborn screening (NBS); designer, mutation specific treatments (Ivacaftor, Lumacaftor) are becoming available, and the first ever Gene Therapy trial (does it help, rather than can it be made to happen) will report later this year (see page 962). The treatment paradigm is changing from firefighting the downstream complications of the disease in sick patients to correction of the fundamental molecular defect in the virtually asymptomatic. But are we victims of our own success? Yes, say the London Poms, lung function (see page 910, Hot topic) and CT scans are so good a year after NBS CF diagnosis that huge studies will be needed to demonstrate any improvement over standard care. No, say the AREST-CF group in Australia, both function and structure are so rubbish that new treatments are urgently needed. Piggy in the middle, editorialist Peter Merkus tries to make sense of these disparate results …
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