Results 1 to 10 of about 1,231,622 (330)

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

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   +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   +3 more sources

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

Front Pharmacol, 2023
The third Sustainable Development Goal (SDG), to ensure healthy lives and promote well-being for all at all ages, has particular relevance and implementation challenges amongst people living with rare diseases such as cystic fibrosis (CF).
Zampoli M, Morrow BM, Paul G.
europepmc   +2 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

Personalized CFTR Modulator Therapy for G85E and N1303K Homozygous Patients with Cystic Fibrosis. [PDF]

Int J Mol Sci, 2023
CFTR modulator therapy with elexacaftor/tezacaftor/ivacaftor (ETI) has been approved for people with CF and at least one F508del allele in Europe. In the US, the ETI label has been expanded to 177 rare CFTR mutations responsive in Fischer rat thyroid ...
Graeber SY, Balázs A, Ziegahn N, Rubil T, Vitzthum C, Piehler L, Drescher M, Seidel K, Rohrbach A, Röhmel J, Thee S, Duerr J, Mall MA, Stahl M.   +13 more
europepmc   +2 more sources

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis. [PDF]

Pharmaceuticals (Basel), 2023
Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly
Bacalhau M, Camargo M, Magalhães-Ghiotto GAV, Drumond S, Castelletti CHM, Lopes-Pacheco M.   +5 more
europepmc   +2 more sources

The gut-lung axis in the CFTR modulator era. [PDF]

Front Cell Infect Microbiol, 2023
The advent of CFTR modulators represents a turning point in the history of cystic fibrosis (CF) management, changing profoundly the disease’s clinical course by improving mucosal hydration.
Lussac-Sorton F, Charpentier É, Imbert S, Lefranc M, Bui S, Fayon M, Berger P, Enaud R, Delhaes L.   +8 more
europepmc   +2 more sources

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

Eur Arch Otorhinolaryngol, 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 ...
Bode SFN, Rapp H, Lienert N, Appel H, Fabricius D.   +4 more
europepmc   +2 more sources

Evolving nutrition therapy in cystic fibrosis: Adapting to the CFTR modulator era. [PDF]

Nutr Clin Pract
Cystic fibrosis transmembrane regulator (CFTR)–directed therapies, such as modulators, have transformed the medical management of people with CF, resulting in better lung function, weight, and body mass index in recent years.
Vavrina K, Griffin TB, Jones AM, Schindler T, Bui TN, Sankararaman S.   +5 more
europepmc   +2 more sources

Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass. [PDF]

Front Pharmacol, 2023
Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. The mechanism as well as the long-term impact of ivacaftor on weight, resting energy expenditure (REE) and body composition
Mouzaki M, Dupuis A, Avolio J, Griffin K, Ratjen F, Tullis E, Gonska T.   +6 more
europepmc   +2 more sources