Article
Ivacaftor for the p.Ser549Arg (S549R) gating mutation - The Israeli experience.
Respiratory medicine - 1 Oct 2017
Dagan Adi, Cohen-Cymberknoh Malena, Shteinberg Michal, Levine Hagit, Vilozni Daphna, Bezalel Yael, Bar Aluma Bat-El, Sarouk Ifat, Ashkenazi Moshe, Lavie Moran, Tsabari Reuven, Blau Hannah, Kerem Eitan, Bentur Lea, Efrati Ori, Livnat Galit
Abstract excerpt
BACKGROUND: Ivacaftor is a drug that increases the probability of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel remaining open. Information about the efficacy of ivacaftor in patients carrying the rare p.Ser549Arg (S549R) CFTR mutation is sparse. AIM: Efficacy of ivacaftor treatment in patients carrying the p.Ser549Arg (S549R) CFTR mutation. METHODS: Data obtained from CF...
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