Results 1 to 10 of about 3,103 (172)

Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis [PDF]

CPT: Pharmacometrics & Systems Pharmacology
Ivacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF ...
Ngoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, Eric Deneuville, Anne Mornand, Philippe Reix, Mélanie Ribault, Pierre‐Régis Burgel, Léo Froelicher‐Bournaud, Steeve Rouillon, Mohammed Rohi Sanoufi, Gabrielle Lui, Saïk Urien, Jean‐Marc Tréluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, Modul‐CF study group   +16 more
doaj   +3 more sources

Management of Infants Exposed to Prenatal and Postnatal Elexacaftor/Tezacaftor/Ivacaftor: A Cross-Sectional Survey. [PDF]

Health Sci Rep
Health Science Reports, Volume 9, Issue 5, May 2026.
Haskett H, Ren CL.
europepmc   +2 more sources

Improved Quality of Life in Children With Cystic Fibrosis Who Received Transmembrane Conductance Regulator Modulators. [PDF]

Acta Paediatr
ABSTRACT Aim Children with cystic fibrosis (CF) face substantial daily treatment burdens and the effects of transmembrane conductance regulator modulators on these have not been sufficiently described. We evaluated changes in treatment burden after elexacaftor tezacaftor ivacaftor (ETI) was initiated.
Svedberg M, Lundqvist F, Östrand HA, Krantz C, Ekman P, Imberg H.   +5 more
europepmc   +2 more sources

Long-term impact of Elexacaftor/Tezacaftor/ivacaftor on pulmonary, nutritional and metabolic outcomes in homozygous F508del cystic fibrosis patients: A real-world cohort study. [PDF]

Br J Clin Pharmacol
Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Perrotta N, Fiorito LA, Vescovo R, Virgilio A, Amato G, Cimino G.   +5 more
europepmc   +2 more sources

Colorectal Cancer Complicated by Pulmonary Embolism and Potential Takotsubo Cardiomyopathy in a Person With Cystic Fibrosis: Diagnostic and Management Considerations. [PDF]

Respirol Case Rep
We describe a complex case of pulmonary embolism and dilated cardiomyopathy in a person with cystic fibrosis. These late complications occurred following laparoscopic left hemicolectomy for colonic adenocarcinoma. This case highlights the complexities of evaluating breathlessness in severe lung disease, and the management challenges that arise as life ...
Frederiksen L, Brown J, Moore V, Reid DW.   +3 more
europepmc   +2 more sources

Elexacaftor-Tezacaftor-Ivacaftor Reduces Revision Sinus Surgery in People With Cystic Fibrosis. [PDF]

Laryngoscope
Elexacaftor–tezacaftor–ivacaftor (ETI) was associated with a significant decrease in the frequency and rate of endoscopic sinus surgeries in patients with cystic fibrosis. These findings suggest an improvement in CF‐related chronic rhinosinusitis outcomes following ETI and may influence future CF treatment decisions.
Lin A, Komara M, Kistler I, Tan ZH, Lind M, Kotha K, Chiang T.   +6 more
europepmc   +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, Alexandra Kavvalou, Matthias Welsner, Raphael Hirtz, Svenja Straßburg, Sivagurunathan Sutharsan, Florian Stehling, Mathis Steindor   +7 more
doaj   +1 more source

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

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, Clémence Martin, Espérie Burnet, Jennifer Da Silva, Pierre-Régis Burgel   +4 more
doaj   +1 more source

Simultaneous Quantification of Ivacaftor, Tezacaftor, and Elexacaftor in Cystic Fibrosis Patients’ Plasma by a Novel LC–MS/MS Method

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, Alessia Cafaro, Manuela Stella, Giammarco Baiardi, Sebastiano Barco, Nicoletta Pedemonte, Claudia D’Orsi, Federico Cresta, Rosaria Casciaro, Carlo Castellani, Maria Grazia Calevo, Francesca Mattioli, Giuliana Cangemi   +12 more
doaj   +1 more source