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Rethinking CFTR variant responsiveness : differential responses to vanzacaftor and elexacaftor
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- Journal Article
- A1
- open access
Machine learning–assisted diagnosis classification of primary immune dysregulation using IDDA2.1 phenotype profiling
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Correlation between patient-derived intestinal organoids and clinical responses to CFTR modulators in people with cystic fibrosis homozygous for F508del
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- Journal Article
- A1
- open access
Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103) : results from two randomised, active-controlled, phase 3 trials
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Airway remodeling in cystic fibrosis is heterogeneous
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Rectal organoid morphology analysis (ROMA) as a novel physiological assay for diagnostic classification in cystic fibrosis
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- Journal Article
- A1
- open access
Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids
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Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR : a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial
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- Journal Article
- A1
- open access
Coronavirus disease 2019 in patients with inborn errors of immunity : an international study
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Chest CT scoring for evaluation of lung sequelae in congenital diaphragmatic hernia survivors