Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis
Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are small-molecule therapeutics that target the underlying cause of CF. Ivacaftor, a CFTR potentiator, improves CFTR function by increasing channel gating activity at the cell surface [1,2]. In clinical trials and real-world studies, ivacaftor has been shown to be safe and efficacious in children as young as 4 months of age with CFTR gating pathogenic variants, with early and sustained improvements in lung function, CFTR function, and respiratory symptoms [3 –6].
Source: Journal of Cystic Fibrosis - Category: Respiratory Medicine Authors: Paul McNally, Alvin Singh, Susanna A. McColley, Jane C. Davies, Mark Higgins, Meng Liu, Jennifer Lu, Violeta Rodriguez-Romero, Judy L. Shih, Margaret Rosenfeld, VX15-770-124 Study Group Tags: Original Article Source Type: research