Results 61 to 70 of about 1,208,918 (256)

Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]

, 2020
Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
core   +1 more source

Advances in bronchiectasis:endotyping, genetics, microbiome, and disease heterogeneity [PDF]

, 2018
Bronchiectasis is characterised by pathological dilation of the airways. More specifically, the radiographic demonstration of airway enlargement is the common feature of a heterogeneous set of conditions and clinical presentations.
Chalmers, James D., Flume, Patrick A., Olivier, Kenneth N.   +2 more
core   +2 more sources

CGM patterns in adults with cystic fibrosis-related diabetes before and after elexacaftor-tezacaftor-ivacaftor therapy

Journal of Clinical & Translational Endocrinology, 2022
Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis that is associated with worse outcomes and higher mortality rates.
Hanna Crow, Charles Bengtson, Xiaosong Shi, Leland Graves, III, Abeer Anabtawi   +4 more
doaj   +1 more source

The Impact of Highly Effective CFTR Modulators on Growth and Nutrition Status

Nutrients, 2021
Patients with cystic fibrosis (CF) are at increased risk of malnutrition and growth failure due to multiple factors as a result of suboptimal or absent function of the CFTR chloride channel protein.
R. Bass, J. Brownell, V. Stallings
semanticscholar   +1 more source

CFTR modulator use and risk of nontuberculous mycobacteria positivity in cystic fibrosis, 2011–2018

ERJ Open Research, 2022
Background People with cystic fibrosis are at increased risk of pulmonary nontuberculous mycobacteria (NTM) disease. Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are associated with reduced lung infection with pathogens like ...
Emily E. Ricotta, D. Rebecca Prevots, Kenneth N. Olivier   +2 more
doaj   +1 more source

New drugs, new challenges in cystic fibrosis care

European Respiratory Review
Cystic fibrosis (CF) is a genetic disease caused by variants in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein, a chloride and bicarbonate channel.
Isabelle Fajac, Pierre-Régis Burgel, Clémence Martin   +2 more
doaj   +1 more source

Non‐pulmonary CFTR‐related symptom improvement with ivacaftor in p.Phe508del/p.Arg117His (7T) cystic fibrosis

Respirology Case Reports, 2023
Diagnosis and management of CRMS/CFSPID and cystic fibrosis (CF) with mild phenotypes remains challenging, and this extends to expanding practice with the use of CFTR modulators.
Stephanie L. Kuek, R. John H. Massie
doaj   +1 more source

Impact of CFTR Modulators on Beta-Cell Function in Children and Young Adults with Cystic Fibrosis

Journal of Clinical Medicine, 2022
Background: To date, no consistent data are available on the possible impact of CFTR modulators on glucose metabolism. The aim of this study was to test the hypothesis that treatment with CFTR modulators is associated with an improvement in the key ...
C. Piona, E. Mozzillo, A. Tosco, S. Volpi, F. Rosanio, C. Cimbalo, A. Franzese, V. Raia, C. Zusi, Federica Emiliani, M. Boselli, M. Trombetta, R. Bonadonna, M. Cipolli, C. Maffeis   +14 more
semanticscholar   +1 more source

Ligand-based virtual-screening identified a novel CFTR ligand which improves the defective cell surface expression of misfolded ABC transporters

Frontiers in Pharmacology
Cystic fibrosis (CF) is a monogenetic disease caused by the mutation of CFTR, a cAMP-regulated Cl− channel expressing at the apical plasma membrane (PM) of epithelia.
Shogo Taniguchi, Francois Berenger, Yukako Doi, Ayana Mimura, Yoshihiro Yamanishi, Tsukasa Okiyoneda   +5 more
doaj   +1 more source

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators

Scientific Reports, 2017
Clinical studies with modulators of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein have demonstrated that functional restoration of the mutated CFTR can lead to substantial clinical benefit.
Iwona M. Pranke, Aurélie Hatton, Juliette Simonin, Jean Philippe Jais, Françoise Le Pimpec-Barthes, Ania Carsin, Pierre Bonnette, Michael Fayon, Nathalie Stremler-Le Bel, Dominique Grenet, Matthieu Thumerel, Julie Mazenq, Valerie Urbach, Myriam Mesbahi, Emanuelle Girodon-Boulandet, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet-Gaudelus   +17 more
doaj   +1 more source