Article
CFTR-dependent chloride efflux in cystic fibrosis mononuclear cells is increased by ivacaftor therapy.
Pediatric pulmonology - 1 Jul 2017
Guerra Lorenzo, D'Oria Susanna, Favia Maria, Castellani Stefano, Santostasi Teresa, Polizzi Angela M, Mariggiò Maria A, Gallo Crescenzio, Casavola Valeria, Montemurro Pasqualina, Leonetti Giuseppina, Manca Antonio, Conese Massimo
Abstract excerpt
AIM: The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiator ivacaftor (Kalydeco®) improves clinical outcome in G551D cystic fibrosis (CF) patients. Here, we have investigated whether ivacaftor has a clinical impact on non-G551D gating mutations and function of circulating leukocytes as well. METHODS: Seven patients were treated with ivacaftor and evaluated at baseline, and at 1-3 and 6 months....
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