Results 81 to 90 of about 3,864 (165)

Home Microbiological Sampling in a Pediatric Cystic Fibrosis Population: Pandemic Implementation and Ongoing Use

Pediatric Pulmonology, Volume 60, Issue 11, November 2025.
ABSTRACT Background During the coronavirus disease 2019 (COVID19) pandemic, restrictions on in‐person care threatened to disrupt in‐clinic airway sampling for microbiological surveillance, a vital aspect of cystic fibrosis (CF) care. In response, institutions developed home airway sampling strategies to allow continued guidelines‐based microbiological ...
Valerie Swanston, Jeffrey N. Bone, Melissa Richmond, David Goldfarb, Jonathan H. Rayment   +4 more
wiley   +1 more source

P323 Does elexacaftor/tezacaftor/ivacaftor triple therapy significantly raise blood lipids? A longitudinal cohort study [PDF]

, 2023
R J Docherty, J. Still, Owen Dempsey
openalex   +1 more source

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor‐Mediated Drug‐Induced Liver Injury Using a Liver‐On‐Chip Model

Clinical and Translational Science, Volume 18, Issue 11, November 2025.
ABSTRACT Elexacaftor/tezacaftor/ivacaftor (ETI), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy, has provided great improvements in lung function and well‐being for people with CF. The use of ETI has been complicated by reports of rare but significant liver function test elevations in clinical trials and drug‐induced ...
Alan Shi, Palak Agarwal, Zhang‐Xu Liu, Paul M. Beringer   +3 more
wiley   +1 more source

Divergent neurobehavioral effects of CFTR modulators elexacaftor and ivacaftor in mice

Acta Psychologica
Recent advances in cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies have markedly improved survival and quality of life for people with cystic fibrosis (CF).
Qian Ge, Amy Fagan, Hui Lu, Jianyang Du, Leah R. Reznikov   +4 more
doaj   +1 more source

Real‐world population pharmacokinetics of tezacaftor‐ivacaftor in children with cystic fibrosis: The SYM‐CF study

British Journal of Clinical Pharmacology, Volume 91, Issue 10, Page 2969-2978, October 2025.
Aims The clinical effectiveness of tezacaftor‐ivacaftor in children with cystic fibrosis (cwCF) varies; some patients respond while others do not or have adverse effects. The pharmacokinetics (PK) of tezacaftor‐ivacaftor are inadequately published, especially in children.
Steffie E. M. Vonk, Suzanne W. J. Terheggen‐Lagro, Eric G. Haarman, Hettie M. Janssens, Anke‐Hilse Maitland–van der Zee, E. Marleen Kemper, Ron A. A. Mathôt, on behalf of the Amsterdam Mucociliary Clearance Disease (AMCD) research group   +7 more
wiley   +1 more source

Deciphering pediatric respiratory diseases using single-cell RNA sequencing [PDF]

The respiratory system comprises specialized cell types that maintain homeostasis and facilitate air conductance and gas exchange. Disease states often involve dysregulation of this balance, leading to changes in cell composition and molecular profiles ...
Loske, Jennifer
core   +1 more source

Functional Pulmonary Imaging

Journal of Magnetic Resonance Imaging, Volume 62, Issue 4, Page 986-1008, October 2025.
ABSTRACT The aging of the world population gave rise to an increased prevalence of many lung diseases, with chronic obstructive pulmonary disease now ranking as the third‐leading cause of death according to the World Health Organization. To diagnose lung disease, a thorough assessment of lung function is essential since it may reveal unique signatures ...
Agilo L. Kern, Filip Klimeš, Andreas Voskrebenzev, Hoen‐Oh Shin, Jens Vogel‐Claussen   +4 more
wiley   +1 more source

Quantification of Ivacaftor, Tezacaftor, Elexacaftor, and Lumacaftor and their active metabolites in plasma using UHPLC-MS/MS:Doors open to the application of therapeutic drug monitoring in cystic fibrosis treatment [PDF]

An ultra-high performance liquid chromatography-tandem mass spectrometry method was developed to quantify the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, tezacaftor, elexacaftor, and lumacaftor and their active ...
Akkerman, Onno W, Elzinga, Femke A, Koppelman, Gerard H, Mian, Paola, Touw, Daan J, Wessels, A Mireille A   +5 more
core   +1 more source

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Filipa C. Ferreira, Camilla D. Buarque, Miquéias Lopes-Pacheco   +2 more
doaj   +1 more source

Phenotype‐Genotype Correlations in ABCA3 Patients—The RespiRare Cohort

Pediatric Pulmonology, Volume 60, Issue 10, October 2025.
ABSTRACT Background ATP‐binding cassette transporter A3 (ABCA3) deficiency is one of the most severe causes of childhood interstitial lung diseases (chILD). This study aims to report the RespiRare ABCA3 cohort and to establish phenotype‐genotype correlations. Methods Phenotypic and genotypic data of patients under 18 years were retrospectively included
Manon Fleury, Céline Delestrain, Alice Hadchouel, Julie Mazenq, Myriam Benhamida, Anne‐Sophie Bernard, Raphaël Borie, Jacques Brouard, Harriet Corvol, Pierrick Cros, Christophe Delacourt, Tifenn Desroziers, Jean‐Christophe Dubus, Carole Egron, Ralph Epaud, Michael Fayon, Aude Forgeron, Lisa Giovannini‐Chami, Christophe Marguet, Alexandra Masson‐Rouchaud, Hortense Petat, Marie‐Catherine Renoux, Léa Roditis, Caroline Thumerelle, Clémentine Vigier, Aurore Coulomb L'Herminé, Hubert Ducou le Pointe, Pascale Fanen, Camille Fletcher, Chiara Sileo, Laureline Berteloot, Camille Louvrier, Alix de Becdelièvre, Marie Legendre, Nadia Nathan   +34 more
wiley   +1 more source