Treatment of Cystic Fibrosis Patients Homozygous for <i>F508del</i> with Lumacaftor-Ivacaftor (Orkambi^®) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells.

Compared with starting levels (122.9 ± 3.2 mEq/L (mean ± SEM)), sweat chloride showed a decreasing trend over time, with a significant difference at six months ( p < 0.05), while BMI and FEV 1 % increased in time, although only by trend.

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