Cystic Fibrosis

Illustration of a mucus cell

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For the first time, cystic fibrosis patients may have a drug that treats the underlying cause of their disease rather than just its symptoms. According to a new clinical trial, the experimental drug helped patients breathe easier, gain healthy weight and cut infections.

Patients taking the new drug, called ivacaftor, showed improvements in lung function and other measures of health two weeks after starting therapy. They also reported feeling a lot better day to day. Those benefits persisted through 48 weeks of the trial, according to datapublished Thursday in the New England Journal of Medicine.