Results 1 to 10 of about 1,208,918 (256)

Mutual Effects of Single and Combined CFTR Modulators and Bacterial Infection in Cystic Fibrosis [PDF]

Microbiology Spectrum, 2023
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators improve clinical outcomes with varied efficacies in patients with CF. However, the mutual effects of CFTR modulators and bacterial adaptation, together with antibiotic regimens, can ...
Cristina Cigana, Ruggero Giannella, Alice Colavolpe, Beatriz Alcalá-Franco, Giulia Mancini, Francesca Colombi, Chiara Bigogno, Ulla Bastrup, Giovanni Bertoni, Alessandra Bragonzi   +9 more
doaj   +4 more sources

Comparative effects of CFTR modulators on phagocytic, metabolic and inflammatory profiles of CF and nonCF macrophages [PDF]

Scientific Reports, 2023
Macrophage dysfunction has been well-described in Cystic Fibrosis (CF) and may contribute to bacterial persistence in the lung. Whether CF macrophage dysfunction is related directly to Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) in ...
Daniel S. Aridgides, Diane L. Mellinger, Lorraine L. Gwilt, Thomas H. Hampton, Dallas L. Mould, Deborah A. Hogan, Alix Ashare   +6 more
doaj   +3 more sources

Medication adherence to CFTR modulators in patients with cystic fibrosis: a systematic review [PDF]

European Respiratory Review
Background In the last decade, a fundamental shift in the treatment of cystic fibrosis (CF) took place due to the introduction of CF transmembrane conductance regulator (CFTR) modulators.
Carina M.E. Hansen, Anna J. Breukelman, Patricia M.L.A. van den Bemt, Annelies M. Zwitserloot, Liset van Dijk, Job F.M. van Boven   +5 more
doaj   +3 more sources

Impact of CFTR modulators on exercise capacity in adolescents with cystic fibrosis [PDF]

ERJ Open Research
Background Exercise capacity is an independent predictor of clinical worsening in cystic fibrosis (CF). There is limited evidence of the impact of cystic fibrosis transmembrane conductance regulator (CFTR) modulators on exercise capacity in children with
Molla Imaduddin Ahmed, Naomi Dayman, Natalie Blyth, Joe Madge, Erol Gaillard   +4 more
doaj   +3 more sources

Personalized medicine: Function of CFTR variant p.Arg334Trp is rescued by currently available CFTR modulators [PDF]

Frontiers in Molecular Biosciences, 2023
Most of the 2,100 CFTR gene variants reported to date are still unknown in terms of their disease liability in Cystic Fibrosis (CF) and their molecular and cellular mechanism that leads to CFTR dysfunction.
Violeta Railean, Cláudia S. Rodrigues, Sofia S. Ramalho, Iris A. L. Silva, Jan Bartosch, Carlos M. Farinha, Ines Pankonien, Margarida D. Amaral   +7 more
doaj   +3 more sources

CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine

Frontiers in Pharmacology, 2020
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR mRNA and protein expression, function, stability or a combination of these.
Miqueias Lopes-Pacheco
exaly   +4 more sources

Unraveling the Mechanism of Action, Binding Sites, and Therapeutic Advances of CFTR Modulators: A Narrative Review [PDF]

Current Issues in Molecular Biology
Cystic fibrosis (CF) is a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride and bicarbonate channel localized on the plasma membrane of epithelial cells.
Debora Baroni
doaj   +3 more sources

Novel CFTR modulator combinations maximise rescue of G85E and N1303K in rectal organoids [PDF]

ERJ Open Research, 2022
Introduction Cystic fibrosis (CF) is a severe monogenic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Marjolein M. Ensinck, Liesbeth De Keersmaecker, Anabela S. Ramalho, Senne Cuyx, Stephanie Van Biervliet, Lieven Dupont, Frauke Christ, Zeger Debyser, François Vermeulen, Marianne S. Carlon   +9 more
doaj   +2 more sources

The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis

Orphanet Journal of Rare Diseases, 2022
Background Over the past decade, a new class of drugs called CFTR (cystic fibrosis transmembrane conductance regulator) modulators have shown to be able to improve clinical outcomes in patient with Cystic Fibrosis.
Enrico Costa, Silvia Girotti, Francesca Pauro, Hubert G. M. Leufkens, Marco Cipolli   +4 more
doaj   +2 more sources

Role of inhaled antibiotics in the era of highly effective CFTR modulators. [PDF]

Eur Respir Rev, 2023
Recurrent and chronic bacterial infections are common in people with cystic fibrosis (CF) and contribute to lung function decline. Antibiotics are the mainstay in the treatment of exacerbations and chronic bacterial infection in CF.
Elborn JS, Blasi F, Burgel PR, Peckham D.   +3 more
europepmc   +2 more sources