Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators
Cells, 2021 Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene encoding the CFTR protein, a chloride channel expressed in many epithelial cells.
Isabelle Fajac
exaly +5 more sources
Unraveling the Mechanism of Action, Binding Sites, and Therapeutic Advances of CFTR Modulators: A Narrative Review [PDF]
Current Issues in Molecular BiologyCystic 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 +4 more sources
CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France
Cells, 2022 Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death ...
Lucile RÉGARD +2 more
exaly +5 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 M Goetz, Adrienne P Savant
exaly +4 more sources
CFTR Modulators Counteract F508del CFTR Functional Defects in a Pancreatic Epithelial Model of Cystic Fibrosis [PDF]
LifeCystic fibrosis is a multisystem disorder caused by mutations in the CFTR gene that lead to impaired ion and fluid transport across secretory epithelia.
Alessandra Ludovico, Debora Baroni
doaj +3 more sources
Impact of Airway Inflammation on the Efficacy of CFTR Modulators [PDF]
Cells, 2021 Defective CFTR biogenesis and activity in cystic fibrosis airways leads to airway dehydration and impaired mucociliary clearance, resulting in chronic airway infection and inflammation.
Carla M. P. Ribeiro, Martina Gentzsch
doaj +4 more sources
Treatment with CFTR Modulators for Cystic Fibrosis: What a Pediatric Gastroenterologist Needs to Know [PDF]
ChildrenBackground: Cystic fibrosis (CF) is a multisystemic disorder caused by CFTR gene mutations, leading to impaired protein function and affecting pulmonary, gastrointestinal, hepatobiliary, skeletal, and nutritional health.
David Gonzalez Jimenez +3 more
doaj +3 more sources
Exposure to CFTR Modulators During Pregnancy in Cystic Fibrosis: Four Cases to Highlight Neonatal Diagnostic Challenges and Outcomes [PDF]
International Journal of Neonatal ScreeningCFTR modulators have transformed the clinical evolution of patients with CF. The number of pregnancies is increasing in women with CF, most of whom are now treated with CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) or Tezacaftor ...
Louis Domenach +11 more
doaj +2 more sources
Cystic Fibrosis and CFTR Modulators: The Impact on Bone Density, Muscle Mass and Strength in Children and Young Adolescents [PDF]
ChildrenBackground/Objectives: Cystic fibrosis (CF) is a multisystemic and genetic disorder. Mutations in the CF transmembrane conductance regulator (CFTR) gene impair the function of the CFTR protein, leading to various complications in multiple organs, mainly ...
Katerina Iordanidou +6 more
doaj +2 more sources
The gut-lung axis in the CFTR modulator era
Frontiers in Cellular and Infection Microbiology, 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.
Florian Lussac-Sorton +22 more
doaj +3 more sources