Results 1 to 10 of about 9,635 (207)

Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer [PDF]

Therapeutic Advances in Medical Oncology
Background: Drug repurposing has emerged as an effective strategy to accelerate drug discovery. Using the pipeline established from a large collaborative drug repurposing project focused on high-grade serous ovarian cancer (HGSOC), we identified ...
Dongli Liu, Michelle Wong-Brown, Farhana Amy Sarker, Bayley Matthews, Jessica Morrison, Kristie-Ann Dickson, Amani Alghalayini, Jana Stojanova, King Man Wan, Jennifer Duggan, Christine Loo, Gill Stannard, Elyse Powell, Brigitte Hodder, Ellen Barlow, Deborah J. Marsh, Caroline E. Ford, Nikola A. Bowden   +17 more
doaj   +2 more sources

Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data.

PLoS ONE, 2023
ObjectivesCystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific gene mutations ...
Lisa J McGarry, Zahra Bhaiwala, Andrea Lopez, Conor Chandler, Christopher G Pelligra, Jaime L Rubin, Theodore G Liou   +6 more
doaj   +3 more sources

Insights Into Patient Variability During Ivacaftor-Lumacaftor Therapy in Cystic Fibrosis

Frontiers in Pharmacology, 2021
Background: The advent of cystic fibrosis transmembrane conductance regulator protein (CFTR) modulators like ivacaftor have revolutionised the treatment of cystic fibrosis (CF).
Patrick O. Hanafin, Isabelle Sermet-Gaudelus, Matthias Griese, Matthias Kappler, Helmut Ellemunter, Carsten Schwarz, Carsten Schwarz, John Wilson, John Wilson, Marsha Tan, Tony Velkov, Gauri G. Rao, Elena K. Schneider-Futschik   +12 more
doaj   +1 more source

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

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

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

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

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