Restriction lift date: 2022-09-30
Clinical outcomes of real-world Kalydeco (ivacaftor); Cork study - understanding the implications of CFTR modulation
Ronan, Nicola J.
University College Cork
CF represents one of the success stories of modern medicine with sustained incremental increases in survival from one of childhood death to one of adulthood survival. Traditionally the management of CF has focused on treating the consequences of CFTR dysfunction. The advent of ivacaftor offers the opportunity to evaluate the effect of restoration of CFTR function on CF lung disease and extrapulmonary manifestations. Global prevalence of the G551D mutation is 4%. Prevalance at Cork CF centre is 23%. Thus this work evaluates the effect of CFTR modulation on clinical parameters, Chest CT, lung microbiota, gut microbiota, gut inflammation, pancreatic function and benefit to the individual patient.
CFTR modulation , Cystic fibrosis , Ivacaftor
Ronan, N. J. 2020. Clinical outcomes of real-world Kalydeco (ivacaftor); Cork study - understanding the implications of CFTR modulation. PhD Thesis, University College Cork.