Results 11 to 20 of about 12,467 (245)

Quantitative Systems Toxicology Predicts Ivacaftor-Induced Oxidative Stress Contributes to CFTR Modulator Hepatotoxicity. [PDF]

Clin Pharmacol Ther
Cystic fibrosis (CF) is a chronic hereditary disease that affects tens of thousands of people worldwide. The introduction of CFTR modulator therapies such as elexacaftor/tezacaftor/ivacaftor (ETI) has significantly improved the quality of life of people with CF.
Shi A, Cornwell C, Yang K, Beringer PM.
europepmc   +2 more sources

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Frontiers in Pharmacology, 2021
Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare
Khadidja Abdallah, Kris De Boeck, Marc Dooms, Steven Simoens   +3 more
doaj   +1 more source

Impact of Elexacaftor-Tezacaftor-Ivacaftor on Quality of Life in Children With Cystic Fibrosis. [PDF]

Pediatr Pulmonol
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
Kümmerli S, Elviro CF, Regamey N, Mornand A, Faouzi M, Rochat I, Blanchon S.   +6 more
europepmc   +2 more sources

Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis

Hepatology Communications, 2023
The cystic fibrosis (CF) transmembrane conductance regulator corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition.
Steven Levitte, Yonathan Fuchs, Russell Wise, Zachary M. Sellers   +3 more
doaj   +1 more source

Targeted therapies to improve CFTR function in cystic fibrosis [PDF]

, 2015
Cystic fibrosis is the most common genetically determined, life-limiting disorder in populations of European ancestry. The genetic basis of cystic fibrosis is well established to be mutations in the cystic fibrosis transmembrane conductance regulator ...
Brodlie, M, Elborn, JS, Haq, IJ, Roberts, K   +3 more
core   +4 more sources

The validated French CFAbd-Score reveals a lower burden of gastrointestinal symptoms in patients on Elexacaftor/Tezacaftor/Ivacaftor. [PDF]

J Pediatr Gastroenterol Nutr
Abstract Objectives Multiorgan abdominal involvement is a hallmark of Cystic fibrosis (CF). The CFAbd‐Score© is the first CF‐specific gastrointestinal patient reported outcome‐measure (PROM) developed following FDA‐guidelines. The PROM has proved to sensitively differentiate people with CF (pwCF) from healthy controls (HC).
Sermet-Gaudelus I, Sadrieh P, Bonnel AS, Enaud R, Wizla N, Languepin J, Nangui A, Deelawon A, Rave J, Dabelow S, Barucha A, Zagoya C, Duckstein F, Mainz JG.   +13 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

Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis

eLife, 2020
Previously, we showed that serum and monocytes from patients with CF exhibit an enhanced NLRP3-inflammasome signature with increased IL-18, IL-1β, caspase-1 activity and ASC speck release (Scambler et al. eLife 2019).
Heledd H Jarosz-Griffiths, Thomas Scambler, Chi H Wong, Samuel Lara-Reyna, Jonathan Holbrook, Fabio Martinon, Sinisa Savic, Paul Whitaker, Christine Etherington, Giulia Spoletini, Ian Clifton, Anil Mehta, Michael F McDermott, Daniel Peckham   +13 more
doaj   +1 more source

A little CFTR goes a long way: CFTR-dependent sweat secretion from G551D and R117H-5T cystic fibrosis subjects taking ivacaftor. [PDF]

PLoS ONE, 2014
To determine if oral dosing with the CFTR-potentiator ivacaftor (VX-770, Kalydeco) improves CFTR-dependent sweating in CF subjects carrying G551D or R117H-5T mutations, we optically measured sweat secretion from 32-143 individually identified glands in ...
Jessica E Char, Marlene H Wolfe, Hyung-Ju Cho, Il-Ho Park, Jin Hyeok Jeong, Eric Frisbee, Colleen Dunn, Zoe Davies, Carlos Milla, Richard B Moss, Ewart A C Thomas, Jeffrey J Wine   +11 more
doaj   +1 more source

Greenness, Whiteness, and Blueness Assessment With a Novel and Fully Validated HPLC Method for Simultaneous Analysis of Lumacaftor and Ivacaftor in Sweat. [PDF]

Int J Anal Chem
Sweat is an alternative biological fluid to plasma, urine, hair, and saliva, and it is promising for various pharmaceutical research types. Excessive sweating is one of the symptoms of cystic fibrosis, a hereditary disease. In this study, an easy, simple, applicable, and economical HPLC method was proposed for sweat analysis of the lumacaftor/ivacaftor
Levent S, Elriş A, Özcan S, Can NÖ.
europepmc   +2 more sources