Restriction lift date: 2022-09-30
Clinical outcomes of real-world Kalydeco (ivacaftor); Cork study - understanding the implications of CFTR modulation
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Date
2020-09-12
Authors
Ronan, Nicola J.
Journal Title
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Volume Title
Publisher
University College Cork
Published Version
Abstract
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.
Description
Keywords
CFTR modulation , Cystic fibrosis , Ivacaftor
Citation
Ronan, N. J. 2020. Clinical outcomes of real-world Kalydeco (ivacaftor); Cork study - understanding the implications of CFTR modulation. PhD Thesis, University College Cork.