Results 1 to 10 of about 5,299 (208)

CFTR Modulator Therapy for Rare CFTR Mutants

Journal of Respiration, 2022
Cystic fibrosis (CF), the most common genetic disease among the Caucasian population, is caused by mutations in the gene encoding for the CF transmembrane conductance regulator (CFTR), a chloride epithelial channel whose dysfunction results in severe ...
Alessandra Ghigo, Alessandra Murabito, Marco Mergiotti   +2 more
exaly   +4 more sources

Hypoglycaemia after Initiation of CFTR Modulator Therapy in a Cystic Fibrosis Patient without Diabetes [PDF]

Case Reports in Endocrinology, 2023
Introduction. Cystic fibrosis transmembrane regulator (CFTR) modulator therapies improve respiratory function and glycaemic control in patients with cystic fibrosis (CF).
Marie Yskout, Joke Vliebergh, Hakan Bor, Lieven Dupont, Natalie Lorent, Pascal Van Bleyenbergh, Pieter Gillard, Bart Van der Schueren, Ann Mertens, Chantal Mathieu, Roman Vangoitsenhoven   +10 more
doaj   +2 more sources

Changes in the Cystic Fibrosis Airway Microbiome in Response to CFTR Modulator Therapy

Frontiers in Cellular and Infection Microbiology, 2021
The development of CFTR modulator therapies significantly changed the treatment scheme of people with cystic fibrosis. However, CFTR modulator therapy is still a life-long treatment, which is not able to correct the genetic defect and cure the disease ...
Sébastien Boutin, Alexander H Dalpke, Buqing Yi   +2 more
exaly   +3 more sources

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   +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

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes [PDF]

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +2 more sources

Review of CFTR modulators 2020 [PDF]

Pediatric Pulmonology, 2021
AbstractCystic fibrosis transmembrane conductance regulator (CFTR) modulators are small molecules that directly impact the CFTR protein, improving the function of the CFTR chloride and bicarbonate channel. Beginning in 2012 with the Food and Drug Administration approval of the first CFTR modulator, ivacaftor, this class of medications has had largely ...
Danielle Goetz, Adrienne Savant
openaire   +2 more sources

Sinus Disease Grading on Computed Tomography Before and After Modulating Therapy in Adult Patients with Cystic Fibrosis

Journal of the Belgian Society of Radiology, 2022
Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy effects on respiratory function, pulmonary exacerbations and quality of life have been well documented. However, CFTR modulator therapy effects on sinus disease have
Corrado Tagliati, Stefano Pantano, Giuseppe Lanni, Davide Battista, Matteo Marcucci, Marco Fogante, Giulio Argalia, Enrico Paci, Gabriella Lucidi Pressanti, Mingliang Ying, Pietro Ripani   +10 more
doaj   +1 more source

Real-world disparities and ethical considerations with access to CFTR modulator drugs: Mind the gap! [PDF]

Frontiers in Pharmacology, 2023
Marco Zampoli
exaly   +2 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