Results 11 to 20 of about 1,459,532 (261)

Identification of early changes in multiple biomarkers following CFTR modulator initiation in patients with cystic fibrosis [PDF]

Therapeutic Advances in Respiratory Disease
Background: There are currently no early parameters that allow prediction of long-term responses to Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator treatment on an individual level.
Pascal Heer, Clara Fernandez Elviro, Angela Koutsokera, Anne Mornand, Isabelle Rochat, Nicolas Regamey, Sylvain Blanchon   +6 more
doaj   +3 more sources

Chronic rhinosinusitis in the era of CFTR modulator therapy.

Journal of Cystic Fibrosis, 2023
Chronic rhinosinusitis is a common manifestation of CF that is associated with impaired quality of life and can be difficult to treat. CFTR modulator therapy has resulted in significant improvements in lower respiratory and nutritional outcomes for ...
R. Stone, C. Short, Jane C. Davies, P. McNally   +3 more
semanticscholar   +5 more sources

Laboratory Tools to Predict CFTR Modulator Therapy Effectiveness and to Monitor Disease Severity in Cystic Fibrosis. [PDF]

J Pers Med
The implementation of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has been attaining remarkable therapeutic outcomes for CF, a life-threatening autosomal recessive genetic disease. However, there
Bacalhau M, Camargo M, Lopes-Pacheco M.
europepmc   +3 more sources

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

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   +3 more sources

Organic Synthesis and Current Understanding of the Mechanisms of CFTR Modulator Drugs Ivacaftor, Tezacaftor, and Elexacaftor. [PDF]

Molecules
The monogenic rare disease Cystic Fibrosis (CF) is caused by mutations in the gene encoding the CF transmembrane conductance (CFTR) protein, an anion channel expressed at the apical plasma membrane of epithelial cells.
Ferreira FC, Buarque CD, Lopes-Pacheco M.   +2 more
europepmc   +3 more sources

Effects of CFTR-modulator triple therapy on sinunasal symptoms in children and adults with cystic fibrosis

European Archives of Oto-Rhino-Laryngology, 2023
Sinunasal symptoms and chronic rhinusinutitis are common in patients with cystic fibrosis. Cystic fibrosis transmembrane regulator (CFTR) modulators have led to dramatic improvements of respiratory symptoms and quality of life in patients with cystic ...
Sebastian F N Bode, Dorit Fabricius, Fabricius Dorit   +2 more
exaly   +2 more sources

PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy

Journal of Cystic Fibrosis, 2021
Highly effective CFTR modulator drug therapy is increasingly available to those with cystic fibrosis. Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on long-term ...
David P Nichols, Scott H Donaldson, Steven D Freedman   +2 more
exaly   +2 more sources

CFTR Modulator Therapies: Potential Impact on Airway Infections in Cystic Fibrosis

Cells, 2022
Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the gene encoding for the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein, expressed on the apical surface of epithelial cells.
F. Saluzzo, Luca Riberi, B. Messore, N. Loré, I. Esposito, E. Bignamini, V. De Rose   +6 more
semanticscholar   +2 more sources

CFTR Modulator Response in Nasal Organoids Derived from People with Cystic Fibrosis [PDF]

Cells
Despite the progressive extension of CFTR variant eligibility to the triple combination of elexacaftor/tezacaftor/ivacaftor (ETI), most rare CFTR pathogenic variants remain ineligible for CFTR modulators.
Stefania Lo Cicero, Germana Castelli, Aurora Ceci, Anna Maria Cerio, Giovanna Blaconà, Mariarita Virgulti, Sara Allushi, Giovanni Sette, Francesca Spadaro, Felice Amato, Paola Melotti, Claudio Sorio, Giuseppe Cimino, Mauro Biffoni, Marco Lucarelli, Adriana Eramo   +15 more
doaj   +2 more sources

CFTR modulator therapy alters plasma sphingolipid profiles in people with cystic fibrosis

Journal of Cystic Fibrosis, 2022
BACKGROUND Sphingolipids, in particular ceramides, play an important role in the pathogenesis of cystic fibrosis (CF) lung disease. Ceramides seem to be dysregulated in people with CF (PWCF): An elevated ratio of ceramides C16Cer/ C24Cer has been linked ...
Dirk Westhölter, Fabian Schumacher, Svenja Strassburg   +2 more
exaly   +2 more sources