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

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dc.check.infoControlled Access
dc.contributor.advisorPlant, Barry
dc.contributor.advisorEustace, Joe
dc.contributor.authorArooj, Parniya
dc.date.accessioned2025-02-18T14:40:09Z
dc.date.available2025-02-18T14:40:09Z
dc.date.issued2024
dc.date.submitted2024
dc.descriptionControlled Access
dc.description.abstractCystic 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.en
dc.description.statusNot peer revieweden
dc.description.versionAccepted Versionen
dc.format.mimetypeapplication/pdfen
dc.identifier.citationArooj, 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.
dc.identifier.endpage180
dc.identifier.urihttps://hdl.handle.net/10468/17064
dc.language.isoenen
dc.publisherUniversity College Corken
dc.rights© 2024, Parniya Arooj.
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subjectCFTR modulation
dc.subjectCystic fibrosis
dc.subjectLumacaftor-Ivacaftor
dc.titleReal-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
dc.typeDoctoral thesis
dc.type.qualificationlevelDoctoral
dc.type.qualificationnameMD - Doctor of Medicine
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