Results 21 to 30 of about 9,635 (207)
Ivacaftor Inhibits Glioblastoma Stem Cell Maintenance and Tumor Progression
Frontiers in Cell and Developmental Biology, 2021 Glioblastoma (GBM) is the most common and malignant primary brain tumor. Glioblastoma stem cells (GSCs) not only initiate and sustain uncontrolled cell proliferation but also resistant to conventional clinical therapies including temozolomide (TMZ ...
Kun Liu +13 more
doaj +1 more source
Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience
Pulmonary Therapy, 2020 Plain Language Summary We performed a study to better understand the long-term impact of treatment with a drug called ivacaftor for patients with cystic fibrosis (CF).
Mark Higgins +4 more
doaj +1 more source
Genetics and Molecular Biology, 2021 This is a descriptive cross-sectional study that aims to determine the distribution of the CFTR causing variant in a group of patients at a cystic fibrosis (CF) center in southern Brazil, as well as to describe causing variants that are treatable with ...
Eliandra da Silveira Lima +3 more
doaj +1 more source
Modulator Therapy for Cystic Fibrosis: An Exploration of Current Research [PDF]
, 2020 Developing a drug therapy that addresses the root cause of cystic fibrosis (CF) by increasing CFTR protein levels has long been a research challenge. After genetic therapy failed because a suitable delivery system could not be found, researchers began ...
Rombocos, Jessalyn
core +1 more source
The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation : a review [PDF]
, 2016 We reviewed the impact of ivacaftor on Scottish paediatric cystic fibrosis (CF) patients ≥ 6 years of age after 12 months of treatment. Statistically significant improvements in FEV1 and BMI and a reduction in sweat chloride, all comparable with ...
Scottish Paediatric Cystic Fibrosis Managed Clinical Network (SPCFMCN)
core +1 more source
Pulmonary Therapy, 2020 Introduction Mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR) affect the quantity and/or function of CFTR protein reaching the cell surface.
Joseph M. Pilewski +6 more
doaj +1 more source
From CFTR biology toward combinatorial pharmacotherapy:expanded classification of cystic fibrosis mutations [PDF]
, 2016 More than 2000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) have been described that confer a range of molecular cell biological and functional phenotypes.
Avramescu, Radu G. +17 more
core +3 more sources
Frontiers in Physiology, 2021 Altered cholesterol homeostasis in cystic fibrosis patients has been reported, although controversy remains. As a major membrane lipid component, cholesterol modulates the function of multiple ion channels by complicated mechanisms.
Guiying Cui +5 more
doaj +1 more source
The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients.
PLoS ONE, 2020 ImportanceSinonasal symptoms in patients suffering from cystic fibrosis can negatively influence the quality of life and sinuses can be a niche for pathogens causing infection and inflammation leading to a decrease of lung function.
Romee Gostelie +7 more
doaj +1 more source
Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease
European Respiratory Review, 2020 Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation ...
Michal Shteinberg +1 more
doaj +1 more source