Results 281 to 290 of about 1,231,622 (330)

CFTR Modulators: Current Status and Evolving Knowledge

Seminars in Respiratory and Critical Care Medicine, 2022
AbstractIn the past decade, the medical management of people with cystic fibrosis (pwCF) has changed with the development of small molecules that partially restore the function of the defective CF transmembrane conductance regulator (CFTR) protein and are called CFTR modulators.
Lucile, Regard, Clémence, Martin, Jennifer, Da Silva, Pierre-Régis, Burgel   +3 more
openaire   +2 more sources

Effect of Triple Combination CFTR Modulator Therapy on Sleep in Adult Patients with Cystic Fibrosis

Respiration, 2022
Background: Sleep-disordered breathing (SDB) and disturbed sleep are common, often underrecognized, comorbidities in people with cystic fibrosis (pwCF). Objectives: We studied the effect of CFTR triple combination therapy elexacaftor/tezacaftor/ivacaftor
Matthias Welsner, T. Schulte, S. Dietz-Terjung, G. Weinreich, F. Stehling, C. Taube, S. Strassburg, Christoph Schoebel, S. Sutharsan   +8 more
semanticscholar   +1 more source

WNK1 and WNK4 modulate CFTR activity

Biochemical and Biophysical Research Communications, 2007
The cystic fibrosis transmembrane conductance regulator (CFTR) is an ATP-gated chloride channel. WNK kinases are widely expressed modulators of ion transport. WNK1 and WNK4, two WNK kinases that are mutated in familial hyperkalemic hypertension (FHHt), are co-expressed with CFTR in several organs, raising the possibility that WNK kinases might alter ...
Chao-Ling, Yang, Xuehong, Liu, Alex, Paliege, Xiaoman, Zhu, Sebastian, Bachmann, David C, Dawson, David H, Ellison   +6 more
openaire   +2 more sources

CFTR Modulator Therapies for Cystic Fibrosis

Pediatric Allergy, Immunology, and Pulmonology, 2015
The cloning of cystic fibrosis transmembrane conductance regulator (CFTR) set into motion a cascade of discoveries that have helped to reveal the underlying pathophysiologic basis of cystic fibrosis (CF). This discovery and the knowledge that followed have also provided the opportunity to target this basic defect, with the hope of reversing or ...
Aaron T, Trimble, Scott H, Donaldson
openaire   +2 more sources

CFTR modulator drug desensitization: Preserving the hope of long term improvement

Pediatric Pulmonology, 2021
The development of modulator therapy has, for the first time, allowed direct targeting of the underlying cause of cystic fibrosis (CF), the cystic fibrosis transmembrane conductance regulator (CFTR). Patients treated with CFTR modulators have improvement
Kelsey Leonhardt, Elizabeth B Autry, R. Kuhn, M. Wurth   +3 more
semanticscholar   +1 more source

Rectal organoid-guided CFTR modulator therapy restores lung function in a cystic fibrosis patient with the rare 1677delTA/R334W genotype

European Respiratory Journal, 2022
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have revolutionised cystic fibrosis (CF) care, leading to significant improvements in lung function, decreased frequency of pulmonary exacerbations and improved quality of life [1 ...
G. Mitropoulou, N. Brandenberg, S. Hoehnel, C. Ceroni, Z. Balmpouzis, S. Blanchon, G. Dorta, A. Sauty, A. Koutsokera   +8 more
semanticscholar   +1 more source

CFTR Modulator Therapy for Cystic Fibrosis

New England Journal of Medicine, 2017
Cystic fibrosis is a disease of abnormal ion transport through epithelium that results in progressive lung disease as well as the involvement of other organs including the pancreas, gut, and liver. Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR), and inheritance is autosomal ...
openaire   +2 more sources

Matrine modulates HSC70 levels and rescues ΔF508‐CFTR

Journal of Cellular Physiology, 2012
AbstractCystic fibrosis transmembrane conductance regulator (CFTR) is a cAMP‐dependent Cl− channel located in the plasma membrane, and its malfunction results in cystic fibrosis (CF), the most common lethal genetic disease in Caucasians. Most CF patients carry the deletion of Phe508 (ΔF508 mutation); this mutation prevents the delivery of the CFTR to ...
BASILE, ANNA, PASCALE, Maria, FRANCESCHELLI, Silvia, Nieddu E, Mazzei MT, Fossa P, TURCO, Maria Caterina, Mazzei M.   +7 more
openaire   +3 more sources

Triple CFTR Modulator Therapy for Cystic Fibrosis

New England Journal of Medicine, 2018
Cystic fibrosis is one of the most common life-threatening autosomal recessive disorders, affecting approximately 80,000 children and adults worldwide.1 It is caused by mutations that result in deficient or defective function of the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel that is normally present in epithelial ...
openaire   +2 more sources

Impact of interruption of CFTR modulator therapies

Journal of Cystic Fibrosis
Novel drug therapy targeting the defective cystic fibrosis transmembrane conductance regulator protein has the potential to significantly enhance the quality of life for numerous patients with cystic fibrosis. However, in some countries social insurance does not pay for modulators because these drugs are extremely expensive.
Burcu, Capraz Yavuz, Ebru, Yalcin, Halime, Nayir Buyuksahin, Birce, Sunman, Ismail, Guzelkas, Didem, Alboga, Meltem, Akgul Erdal, Havva Ipek, Demir, Raziye, Atan, Nagehan, Emiralioglu, Deniz, Dogru, Ugur, Ozcelik, Nural, Kiper   +12 more
openaire   +2 more sources