Results 31 to 40 of about 12,467 (245)

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, Kun Liu, Jun Pu, Zhi Nie, Zhi Nie, Yulin Shi, Yulin Shi, Liping Jiang, Qisheng Wu, Yongbin Chen, Yongbin Chen, Yongbin Chen, Cuiping Yang, Cuiping Yang   +13 more
doaj   +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

Paediatrics: messages from Munich. [PDF]

, 2015
The aim of this article is to describe paediatric highlights from the 2014 European Respiratory Society (ERS) International Congress in Munich, Germany.
Albuali, Alexopoulos, Andersson, Anton-Pacheco, Arigliani, Arumugam, Balfour-Lynn, Baraldi, Barikbin, Bekhof, Benseler, Berankova, Bilton, Bon, Boon, Boudewyns, Brinkman, Casas, Chiu, Constant, Conway, Dassios, DeBoer, Duijts, Elborn, Engelkes, Engelkes, Everard, Fainardi, Fouzas, Frankenberger, Gadomski, Gilchrist, Goksör, Goussard, Goutaki, Gray, Grosse-Onnebrink, Hatziagorou, Henckel, Hirst, Honková, Iglesias, Iglesias, Ingham, Ioan, Jedynak-Wasowicz, Jung, Kirkby, Kobbernagel, Koschlig, Krivec, Loza, Luciani, Lukkarinen, Madkour, Maguire, Mandadzhieva, Marshall, Maurer, Meurling, Moreno, Moreno, Mulder, Murray, Murray, Mustafayev, Mutti, Naehrig, Ohn, Olsson, Ong, Onland, Parekh, Pie tropaoli, Popovic, Pramana, Ramsey, Rosenow, Rossor, Rubio, Schoegler, Schoegler, Schultz, Serio, Smit, Smyth, Stern, Stokholm, Su, Svedberg, Thouvenin, Tiwari, Umutoglu, Uyan, Van Bragt, Van der Schee, Waddingham, Yacob, Yammine   +99 more
core   +11 more sources

Long-Term Ivacaftor in People Aged 6 Years and Older with Cystic Fibrosis with Ivacaftor-Responsive Mutations

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, Kris De Boeck, Jerry A. Nick, Simon Tian, Cynthia DeSouza, Mark Higgins, Richard B. Moss   +6 more
doaj   +1 more source

Frequency of CFTR variants in southern Brazil and indication for modulators therapy in patients with cystic fibrosis

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, Luíse Sgarabotto Pezzin, Ana Carolina Fensterseifer, Leonardo Araújo Pinto   +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

Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy

Frontiers in Immunology, 2023
IntroductionCystic fibrosis (CF), especially CF lung disease, is characterized by chronic infection, immune dysfunction including impairment of regulatory T cells (Tregs) and an exaggerated inflammatory response.
Dirk Westhölter, Jonas Raspe, Hendrik Uebner, Johannes Pipping, Mona Schmitz, Svenja Straßburg, Sivagurunathan Sutharsan, Matthias Welsner, Christian Taube, Sebastian Reuter   +9 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., Balch, William E., Brodsky, Jeffrey L., Cai, Zhiwei, Chiang, Annette N., Cutting, Garry R., Cyr, Douglas M., Frizzell, Raymond A., Guggino, William B., Hong, Jeong S., Houck, Scott A., Lukacs, Gergely L., Peters, Kathryn W., Pollard, Harvey B., Sheppard, David, Skach, William R., Sorscher, Eric J., Veit, Gudio   +17 more
core   +3 more sources

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. [PDF]

, 2014
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that result in reduced anion conductance at the apical membrane of secretory epithelia.
Apaja, Pirjo M., Avramescu, Radu G., Bagdany, Miklos, Borot, Florence, Finkbeiner, Walter E., Hegedus, Tamas, Lukacs, Gergely L., Perdomo, Doranda, Phuan, Puay-Wah, Szollosi, Daniel, Veit, Guido, Verkman, Alan S., Wu, Yu-Sheng   +12 more
core   +1 more source

Correcting for tissue nitrogen excretion in multiple breath washout measurements. [PDF]

, 2017
Nitrogen excreted from body tissues impacts the calculation of multiple breath nitrogen washout (MBWN2) outcomes. The aim of this study was to determine the effect of tissue N2 on MBWN2 outcomes in both healthy subjects and patients with CF and to assess
Jensen, Renee, Kane, Mica, McDonald, Reginald, Ratjen, Felix, Rayment, Jonathan H., Stanojevic, Sanja   +5 more
core   +8 more sources