Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor [PDF]
Pulmonary Therapy, 2020 Introduction The triple-combination (TC) cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen elexacaftor, tezacaftor, and ivacaftor was shown to be safe and efficacious in phase 3 trials of people with cystic fibrosis (pwCF) ≥ 12
Alice Tsai +6 more
doaj +2 more sources
Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor [PDF]
Frontiers in Pharmacology, 2023 Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF).
Burkhard Tümmler, Burkhard Tümmler
doaj +3 more sources
Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials [PDF]
Lancet Respiratory Medicine,the, 2023 BACKGROUND: Elexacaftor-tezacaftor-ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508del allele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator ...
Jennifer L Taylor-Cousar, Mscs +2 more
exaly +3 more sources
Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysisResearch in context [PDF]
EClinicalMedicineSummary: Background: The development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators (correctors and potentiators) emerged as a promising approach, aiming to restore CFTR protein function. A lack of head-to-head trials comparing
Mohammed Safeer V S +7 more
doaj +2 more sources
Exposure to CFTR Modulators During Pregnancy in Cystic Fibrosis: Four Cases to Highlight Neonatal Diagnostic Challenges and Outcomes [PDF]
International Journal of Neonatal ScreeningCFTR modulators have transformed the clinical evolution of patients with CF. The number of pregnancies is increasing in women with CF, most of whom are now treated with CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) or Tezacaftor ...
Louis Domenach +11 more
doaj +2 more sources
Real-world population pharmacokinetics of tezacaftor-ivacaftor in children with cystic fibrosis: The SYM-CF study. [PDF]
Br J Clin PharmacolAims: The clinical effectiveness of tezacaftor-ivacaftor in children with cystic fibrosis (cwCF) varies; some patients respond while others do not or have adverse effects.
Vonk SEM +7 more
europepmc +3 more sources
People with cystic fibrosis with high sputum neutrophil elastase on elexacaftor-tezacaftor-ivacaftor exhibit worse pulmonary function and pro-inflammatory airway milieu [PDF]
Scientific ReportsCystic fibrosis (CF) is a genetic condition affecting over 100,000 individuals worldwide. Lung disease is the main cause of mortality in CF, with chronic neutrophilic inflammation as a hallmark.
Alexandre Cammarata-Mouchtouris +9 more
doaj +2 more sources
Large variations in total and allele-specific transcript expression in a disease mutation-independent manner [PDF]
Scientific ReportsIndividuals with monogenic diseases, even those with identical disease-causing mutations, exhibit considerable clinical heterogeneity in severity and outcomes.
Moritz Freyberg +13 more
doaj +2 more sources
Cutaneous and Ocular Rosacea Associated with Elexacaftor, Tezacaftor and Ivacaftor, A Treatment for Cystic Fibrosis: A Case Report. [PDF]
J Pediatr Clin PractPiednoir A +4 more
europepmc +2 more sources
Comparison of Stool Microbiome in Children with Cystic Fibrosis Treated with and Without Elexacaftor-Tezacaftor-Ivacaftor-A Pilot Study. [PDF]
Int J Mol SciSankararaman S +9 more
europepmc +3 more sources