Results 31 to 40 of about 1,231,622 (330)

A grumbling concern: an international survey of gastrointestinal symptoms in cystic fibrosis in the modulator era [version 1; peer review: 1 approved, 3 approved with reservations]

NIHR Open Research, 2023
Background: Gastrointestinal symptoms in cystic fibrosis (CF) are common and intrusive to daily life. Relieving gastrointestinal symptoms was identified as an important research priority and previously explored in an international survey in 2018. However,
Vinuja Premakumar, Vigilius Gleetus, Zoe Elliott, Bu Hayee, Nicola J Rowbotham, Bethinn Evans, Helen Barr, Siobhán B Carr, Daniel Peckham, Alexander Horsley, Alan R Smyth, Natalie Goodchild, Rebecca J Calthorpe   +12 more
doaj   +1 more source

Editorial: Real-world experience with CFTR modulator therapy. [PDF]

Front Pharmacol, 2023
Tümmler B, Burgel PR.
europepmc   +4 more sources

Pharmacological Responses of the G542X-CFTR to CFTR Modulators

Frontiers in Molecular Biosciences, 2022
Cystic fibrosis (CF) is a lethal hereditary disease caused by loss-of-function mutations of the chloride channel cystic fibrosis transmembrane conductance regulator (CFTR). With the development of small-molecule CFTR modulators, including correctors that facilitate protein folding and expression and potentiators that promote channel activity, about 90%
Xinxiu Fang, Jiunn-Tyng Yeh, Tzyh-Chang Hwang, Tzyh-Chang Hwang   +3 more
openaire   +3 more sources

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

Measurements of spontaneous CFTR-mediated ion transport without acute channel activation in airway epithelial cultures after modulator exposure

Scientific Reports, 2021
Quantitation of CFTR function in vitro is commonly performed by acutely stimulating then inhibiting ion transport through CFTR and measuring the resulting changes in transepithelial voltage (Vte) and current (ISC).
Heidi J. Nick, Pamela L. Zeitlin, Sangya Yadav, Preston E. Bratcher   +3 more
doaj   +1 more source

Increase of liver stiffness and altered bile acid metabolism after triple CFTR modulator initiation in children and young adults with cystic fibrosis

Liver international (Print), 2023
Novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies (elexacaftor/tezacaftor/ivacaftor—ETI) promise clinically significant and sustained improvements for patients with cystic fibrosis (CF).
A. Schnell, J. Jüngert, D. Klett, Hannah Hober, Natalie Kaiser, R. Ruppel, Annika Geppert, Christina Tremel, Julia Sobel, Erika Plattner, S. Schmitt-Grohé, S. Zirlik, D. Strobel, M. Neurath, F. Knieling, M. Rauh, J. Woelfle, André Hoerning, Adrian P. Regensburger   +18 more
semanticscholar   +1 more source

CFTR modulators: transformative therapies for cystic fibrosis [PDF]

Journal of Managed Care & Specialty Pharmacy, 2021
DISCLOSURES: No funding contributed to the writing of this commentary. Both authors are employed by the Cystic Fibrosis Foundation. The Cystic Fibrosis Foundation has entered into therapeutic development award agreements and licensing agreements to assist with the development of CFTR modulators that may result in intellectual property rights, royalties,
Mary, Dwight, Bruce, Marshall
openaire   +2 more sources

S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy in vivo and in vitro in matched pediatric patient-derived cell models

Frontiers in Pediatrics, 2022
Cystic Fibrosis (CF) results from over 400 different disease-causing mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. These CFTR mutations lead to numerous defects in CFTR protein function.
Katelin M. Allan, Katelin M. Allan, Miro A. Astore, Laura K. Fawcett, Laura K. Fawcett, Laura K. Fawcett, Sharon L. Wong, Sharon L. Wong, Po-Chia Chen, Renate Griffith, Adam Jaffe, Adam Jaffe, Adam Jaffe, Serdar Kuyucak, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters, Shafagh A. Waters   +17 more
doaj   +1 more source

YES1 Kinase Mediates the Membrane Removal of Rescued F508del-CFTR in Airway Cells by Promoting MAPK Pathway Activation via SHC1

Biomolecules, 2023
Recent developments in CFTR modulator drugs have had a significant transformational effect on the treatment of individuals with Cystic Fibrosis (CF) who carry the most frequent F508del-CFTR mutation in at least one allele.
Patrícia Barros, Ana M. Matos, Paulo Matos, Peter Jordan   +3 more
doaj   +1 more source

CFTR Modulates Orai1 Dynamics [PDF]

Biophysical Journal, 2011
Calcium signaling regulates many cellular processes and is upregulated in cystic fibrosis (CF) epithelial cells. Agonists stimulate Ca2+ release from ER stores and trigger the formation of a complex between the plasma membrane (PM) Ca2+ channel Orai1 and stromal interaction molecule 1 (STIM1), a primarily endoplasmic reticulum (ER) resident protein ...
AbuArish, Asmahan, Balghi, Haouaria, Arane, Tal, Wiseman, Paul, Hanrahan, John   +4 more
openaire   +1 more source