Results 11 to 20 of about 7,354 (172)

Real-world population pharmacokinetics of tezacaftor-ivacaftor in children with cystic fibrosis: The SYM-CF study. [PDF]

Br J Clin Pharmacol
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.
Vonk SEM, Terheggen-Lagro SWJ, Haarman EG, Janssens HM, Maitland-van der Zee AH, Kemper EM, Mathôt RAA, Amsterdam Mucociliary Clearance Disease (AMCD) research group.   +7 more
europepmc   +2 more sources

Real-World Evaluation of Outcomes and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Pediatric Patients With Cystic Fibrosis: A Retrospective Study. [PDF]

Clin Transl Sci
ABSTRACT Elexacaftor/Tezacaftor/Ivacaftor (ETI) therapy has significantly improved clinical outcomes in people with cystic fibrosis (PwCF) carrying at least one Phe508del CFTR mutation. However, real‐world evidence on the safety and effectiveness of ETI in pediatric populations remains limited, particularly in children with more severe disease ...
Perrotta N, Fiorito LA, Gentile R, Vescovo R, Piciocchi A, Troiani P, Poscia R, Cimino G.   +7 more
europepmc   +2 more sources

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

The magnitude of ivacaftor effects on fluid secretion via R117H-CFTR channels: Human in vivo measurements.

PLoS ONE, 2017
We optically measured effects of orally available ivacaftor (Kalydeco®) on sweat rates of identified glands in 3 R117H subjects, each having a unique set of additional mutations, and compared them with 5 healthy control subjects tested contemporaneously.
Jessica E Char, Colleen Dunn, Zoe Davies, Carlos Milla, Richard B Moss, Jeffrey J Wine   +5 more
doaj   +1 more source

Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass

Frontiers in Pharmacology, 2023
Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. The mechanism as well as the long-term impact of ivacaftor on weight, resting energy expenditure (REE) and body composition
Marialena Mouzaki, Annie Dupuis, Julie Avolio, Katherine Griffin, Felix Ratjen, Elizabeth Tullis, Tanja Gonska   +6 more
doaj   +1 more source

The potentially beneficial central nervous system activity profile of ivacaftor and its metabolites

ERJ Open Research, 2018
Ivacaftor–lumacaftor and ivacaftor are two new breakthrough cystic fibrosis transmembrane conductance modulators. The interactions of ivacaftor and its two metabolites hydroxymethylivacaftor (iva-M1) and ivacaftorcarboxylate (iva-M6) with ...
Elena K. Schneider, Rachel M. McQuade, Vincenzo C. Carbone, Felisa Reyes-Ortega, John W. Wilson, Brenda Button, Ayame Saito, Daniel P. Poole, Daniel Hoyer, Jian Li, Tony Velkov   +10 more
doaj   +1 more source

Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor

Clinical and Translational Science, 2019
Drug–drug interaction (DDI) studies are described for tezacaftor/ivacaftor, a new cystic fibrosis transmembrane conductance regulator modulator therapy for the treatment of cystic fibrosis.
Varun Garg, Jinshan Shen, Chonghua Li, Sagar Agarwal, Asfiha Gebre, Sarah Robertson, Jiayin Huang, Linda Han, Licong Jiang, Kristin Stephan, Linda T. Wang, Julie Lekstrom‐Himes   +11 more
doaj   +1 more source

Ivacaftor Inhibits Glioblastoma Stem Cell Maintenance and Tumor Progression

Frontiers in Cell and Developmental Biology, 2021
Glioblastoma (GBM) is the most common and malignant primary brain tumor. Glioblastoma stem cells (GSCs) not only initiate and sustain uncontrolled cell proliferation but also resistant to conventional clinical therapies including temozolomide (TMZ ...
Kun Liu, Kun Liu, Jun Pu, Zhi Nie, Zhi Nie, Yulin Shi, Yulin Shi, Liping Jiang, Qisheng Wu, Yongbin Chen, Yongbin Chen, Yongbin Chen, Cuiping Yang, Cuiping Yang   +13 more
doaj   +1 more source

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

Pulmonary Therapy, 2020
Plain Language Summary We performed a study to better understand the long-term impact of treatment with a drug called ivacaftor for patients with cystic fibrosis (CF).
Mark Higgins, Nataliya Volkova, Kristin Moy, Bruce C. Marshall, Diana Bilton   +4 more
doaj   +1 more source