Systematic Review of Nasal Endoscopy Scores in Cystic Fibrosis Patients Treated With Cystic Fibrosis Transmembrane Conductance Regulator Modulators. [PDF]
Pediatr PulmonolABSTRACT Introduction Cystic fibrosis (CF) is a severe genetic disorder caused by pathogenic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, leading to multisystem complications including chronic rhinosinusitis and nasal polyposis. Recent advances in CFTR modulator therapies have revolutionized systemic disease control,
Gallardo R +4 more
europepmc +2 more sources
Vanzacaftor–tezacaftor–deutivacaftor for children aged 6–11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial [PDF]
Lancet Respiratory Medicine,theBACKGROUND: In phase 2 trials in people with cystic fibrosis aged 18 years and older, vanzacaftor–tezacaftor–deutivacaftor has been shown to be a safe and effective, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
Jennifer L Taylor-Cousar, Mscs +2 more
exaly +2 more sources
Real-life impact of highly effective CFTR modulator therapy in children with cystic fibrosis
Frontiers in Pharmacology, 2023 Introduction: Recently, cystic fibrosis transmembrane regulator modulator therapy with elexacaftor/tezacaftor/ivacaftor has become available for children with cystic fibrosis (CF) carrying at least one F508del mutation.Objective: To assess the ...
Margarete Olivier +7 more
doaj +1 more source
Cutaneous Adverse Effects of Elexacaftor-Tezacaftor-Ivacaftor: A Single Center Cohort Study on Acne Severity in Adults With Cystic Fibrosis. [PDF]
J Cosmet DermatolSmith AD +5 more
europepmc +3 more sources
Cystic fibrosis modulator therapy can reverse cystic bronchiectasis
Respirology Case Reports, 2023 Bronchiectasis is often considered progressive and irreversible, so cases of regression or reversal are an important step in understanding the underlying pathophysiological mechanisms.
Peter G. Middleton, Nicholas J. Simmonds
doaj +1 more source
Children, 2023 Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator treatment, used over the last few years, which has shown an improvement in different clinical outcomes in patients with cystic ...
Nikoletta Kapouni +3 more
doaj +1 more source
Modulation of host inflammatory pathways by <i>Pseudomonas aeruginosa</i> extracellular vesicles in cystic fibrosis: impact of pulmonary exacerbation and elexacaftor-tezacaftor-ivacaftor treatment. [PDF]
Front ImmunolSaid M +6 more
europepmc +3 more sources
Impact of COVID-19 and CFTR Modulators on Cystic Fibrosis: A Real-World Analysis of Care Patterns. [PDF]
Pediatr PulmonolABSTRACT Introduction Novel therapeutics and rapid expansion of telehealth have reshaped cystic fibrosis (CF) care; however, the impact on visit patterns and equitable access across the CF population remains unclear. We characterized changes in visit patterns from 2017 to 2022 and the association of sociodemographic and clinical factors with visit ...
Hinton AC +3 more
europepmc +2 more sources
CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France
Cells, 2022 Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death ...
Lucile Regard +4 more
doaj +1 more source
Biomedicines, 2023 The new breakthrough cystic fibrosis (CF) drug combination of ivacaftor (IVA), tezacaftor (TEZ), and elexacaftor (ELX), namely “caftor” drugs, directly modulates the activity and trafficking of the defective CF transmembrane conductance regulator protein
Federica Pigliasco +12 more
doaj +1 more source