Spirometric and anthropometric improvements in response to elexacaftor/tezacaftor/ivacaftor depending on age and lung disease severity [PDF]
, 2023 Introduction: Triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy with elexacaftor/tezacaftor/ivacaftor (ETI) was introduced in August 2020 in Germany for people with CF (pwCF) ≥12 years (yrs.) of age and in ...
Büttner, Tina +14 more
core +1 more source
Thoracic Research and PracticeThe objective of the study was to assess and compare the efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) treatment with TEZ/IVA treatment in individuals diagnosed with cystic fibrosis (CF) and carrying the F508del allele.
Kainat Hussain +3 more
doaj +1 more source
Antibiotics, 2021 The new CFTR modulator combination, elexacaftor/tezacaftor/ivacaftor (Trikafta) was approved by the FDA in October 2019 for treatment of Cystic Fibrosis in patients 6 years of age or older who have at least one F508del mutation in one allele and a ...
Marika Comegna +8 more
doaj +1 more source
Impact of Elexacaftor‐Tezacaftor‐Ivacaftor on Quality of Life in Children With Cystic Fibrosis
Pediatric Pulmonology, Volume 61, Issue 1, January 2026.ABSTRACT Objectives CFTR modulators have revolutionized cystic fibrosis (CF) management by targeting the defective protein rather than its consequences. Their impact on quality of life (QoL) have been studied in numerous trials, but few data are available on QoL in patients receiving Elexacaftor‐Tezacaftor‐Ivacaftor (ETI), notably in children given its
Sara Kümmerli +6 more
wiley +1 more source
A pediatric cystic fibrosis arthropathy case who responded to Elexacaftor/Tezacaftor/Ivacaftor therapy [PDF]
, 2023 Meliksah Arslan +4 more
openalex +1 more source
Perceived burden of respiratory physiotherapy in people with cystic fibrosis taking elexacaftor–tezacaftor–ivacaftor combination: a 1-year observational study [PDF]
Background: To limit the progression of disease, people with cystic fibrosis (pwCF) perform daily respiratory physiotherapy, which is perceived as the most burdensome routine in managing their condition.
Blardone, Chiara +12 more
core +1 more source
The L467F-F508del Complex Allele Hampers Pharmacological Rescue of Mutant CFTR by Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients: The Value of the Ex Vivo Nasal Epithelial Model to Address Non-Responders to CFTR-Modulating Drugs [PDF]
, 2022 open16noLoss-of-function mutations of the CFTR gene cause cystic fibrosis (CF) through a variety of molecular mechanisms involving altered expression, trafficking, and/or activity of the CFTR chloride channel. The most frequent mutation among CF patients,
Baffico A. M. +15 more
core +1 more source
Clinical Transplantation, Volume 40, Issue 1, January 2026.ABSTRACT Background: In pediatric lung transplantation, the ability to estimate the projected waiting time could be particularly helpful for candidates and physicians. A score to predict waiting time has yet to be developed in this field. The purpose of this study was to create a score that predicts waitlist time for pediatric patients who are listed ...
Jose Ramirez +2 more
wiley +1 more source
Elexacaftor-Tezacaftor-Ivacaftor corrects monocyte microbicidal deficiency in cystic fibrosis [PDF]
, 2022 Question. Cystic Fibrosis (CF), which is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), is characterized by chronic bacterial lung infection and inflammation.
Daniele Chiappetta +10 more
core +1 more source
Respiratory Medicine Case Reports, 2022 Elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) is a triple combination drug therapy approved for individuals with cystic fibrosis (CF) who possess at least one copy of the F508del cystic fibrosis transmembrane conductance regulator (CFTR) variant.
Elena Stekolchik +2 more
doaj +1 more source