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oai:cora.ucc.ie:10468/170642025-02-19T02:03:05Zcom_10468_386com_10468_4com_10468_1475com_10468_6com_10468_848com_10468_1col_10468_387col_10468_8039col_10468_1186col_10468_8983

Controlled Access Plant, Barry Eustace, Joe Arooj, Parniya 2025-02-18T14:40:09Z 2025-02-18T14:40:09Z 2024 2024 Controlled Access Cystic fibrosis (CF) stands as one of modern medicine's success stories, with significant and sustained improvements in survival rates, transforming it from a childhood fatal condition to one of adult survival. Historically, CF management has centered on addressing the consequences of CFTR dysfunction. The introduction of Lumacaftor-Ivacaftor marks a pivotal shift, allowing researchers to assess the impact of CFTR function restoration on both pulmonary disease and extrapulmonary manifestations. Globally, approximately 82% of individuals with CF are homozygous for the F508del mutation, whereas this prevalence is 53% at the Cork CF centre. This study examines the effects of CFTR modulation on clinical outcomes, patient-reported measures, systemic and airway inflammation, and lung microbiota composition. Not peer reviewed Accepted Version application/pdf Arooj, P. 2024. Real-world Orkambi Cork (ROCK) study - a prospective 12 months analysis addressing the impact of CFTR modulation in patients with cystic fibrosis homozygous for F508del CFTR variant. MD Thesis, University College Cork. 180 https://hdl.handle.net/10468/17064 en University College Cork © 2024, Parniya Arooj. https://creativecommons.org/licenses/by-nc-nd/4.0/ CFTR modulation Cystic fibrosis Lumacaftor-Ivacaftor Real-world Orkambi Cork (ROCK) study - a prospective 12 months analysis addressing the impact of CFTR modulation in patients with cystic fibrosis homozygous for F508del CFTR variant Doctoral thesis Doctoral MD - Doctor of Medicineinfo:eu-repo/semantics/embargoedAccess