Article
Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor.
Digestive diseases and sciences - 1 Jan 2016
Borowitz Drucy, Lubarsky Barry, Wilschanski Michael, Munck Anne, Gelfond Daniel, Bodewes Frank, Schwarzenberg Sarah Jane
Abstract excerpt
BACKGROUND: The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability. AIM: To further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged ≥6 years with CF and the G551D mutation....
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