Digestive diseases and sciences
-
Randomized Controlled Trial Multicenter Study
Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor.
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. ⋯ Nutritional status improved following treatment with ivacaftor for 48 weeks.