Results 11 to 20 of about 9,288 (216)

Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor

Pulmonary Therapy, 2020
Introduction The triple-combination (TC) cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen elexacaftor, tezacaftor, and ivacaftor was shown to be safe and efficacious in phase 3 trials of people with cystic fibrosis (pwCF) ≥ 12 
Alice Tsai, Shu-Pei Wu, Eric Haseltine, Sanjeev Kumar, Samuel M. Moskowitz, Paul Panorchan, Kushal Shah   +6 more
doaj   +1 more source

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

Elexacaftor/tezacaftor/ivacaftor projected survival and long-term health outcomes in people with cystic fibrosis homozygous for F508del.

Journal of Cystic Fibrosis, 2023
BACKGROUND A series of phase 3 clinical trials have demonstrated that elexacaftor plus tezacaftor plus ivacaftor (ELX/TEZ/IVA) is safe and efficacious in people with cystic fibrosis (pwCF) aged ≥12 years with ≥1 F508del mutation in the cystic fibrosis ...
A. Lopez, Conor Daly, G. Vega-Hernández, G. MacGregor, J. Rubin   +4 more
semanticscholar   +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

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

New England Journal of Medicine, 2019
BACKGROUND Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation.
P. Middleton, M. Mall, P. Dřevínek, L. Lands, E. McKone, Deepika Polineni, B. Ramsey, J. Taylor-Cousar, E. Tullis, F. Vermeulen, G. Marigowda, C. Mckee, S. Moskowitz, N. Nair, J. Savage, C. Simard, S. Tian, D. Waltz, F. Xuan, S. Rowe, R. Jain   +20 more
semanticscholar   +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

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

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

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