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The Lancet. Respiratory Medicine|December 23, 2021
Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trialSivagurunathan Sutharsan, Edward F McKone, Damian G Downey, et al.Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society|January 4, 2025
Sweat chloride reflects CFTR function and correlates with clinical outcomes following CFTR modulator treatmentEdith T Zemanick, Bonnie Ramsey, Dorota Sands, et al.Nature Communications|August 15, 2022
ELF5 is a potential respiratory epithelial cell-specific risk gene for severe COVID-19Maik Pietzner, Robert Lorenz Chua, Eleanor Wheeler, et al.Chest|June 5, 2026
Longitudinal Changes in Lung Morphology and Perfusion Detected by Magnetic Resonance Imaging in Patients with Primary Ciliary Dyskinesia From Infancy Through AdulthoodLena Wucherpfennig, Margherita S Silani, Monika Eichinger, et al.Stem Cell Research & Therapy|October 20, 2025
Human induced pluripotent stem cells for in vitro modeling of impaired mucociliary clearance in cystic fibrosis lung diseaseMark-Christian Klassen, Anita Balázs, Janina Zöllner, et al.The Lancet. Respiratory Medicine|January 5, 2025
Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trialJordana E Hoppe, Ajay S Kasi, Jessica E Pittman, et al.American Journal of Respiratory and Critical Care Medicine|February 25, 2026
Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis and Rare CFTR Variants: In Vitro Translation to a Phase 3, Double-Blind, Randomized, Placebo-controlled Trial and Real-World StudyCarlo Castellani, Pedro Mondejar-Lopez, Fredrick Van Goor, et al.Nucleic Acids Research|October 5, 2017
Homeostatic nuclear RAGE-ATM interaction is essential for efficient DNA repairVarun Kumar, Thomas Fleming, Stefan Terjung, et al.Annals of the American Thoracic Society|March 16, 2026
Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: Results from an open-label extension studyJames F Chmiel, Kimberly McBennett, Bradley S Quon, et al.The New England Journal of Medicine|October 19, 2018
VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del AllelesDominic Keating, Gautham Marigowda, Lucy Burr, et al.Pageof 38