Despite therapeutic effects observed in phase II clinical trials ( Sermet-Gaudelus et al., 2010 ; Wilschanski et al., 2011 ), ataluren treatment only demonstrated a favorable trend, albeit not significant, to improve ppFEV 1 of CF patients carrying a nonsense CFTR mutation in at least one allele in a phase III clinical trial ( Kerem et al., 2014 ).
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CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine.
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