Results 21 to 30 of about 9,731 (210)

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, Nataliya Volkova, Kristin Moy, Bruce C. Marshall, Diana Bilton   +4 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

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

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

A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation [PDF]

, 2011
BACKGROUND: Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis.
Bell, Scott C., Davies, Jane, Dong, Qunming, Drevinek, Pavel, Elborn, J. Stuart, Griese, Matthias, Konstan, Michael W., McElvaney, N. Gerard, McKone, Edward F., Moss, Richard, Ordonez, Claudia, Ramsey, Bonnie W., Ratjen, Felix, Rodriguez, Sally, Rowe, Steven M., Sermet-Gaudelus, Isabelle, Tullis, Elizabeth, Wainwright, Claire E., Yen, Karl   +18 more
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

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

The therapeutic potential of small-molecule modulators of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel [PDF]

, 2014
The cystic fibrosis transmembrane conductance regulator (CFTR) plays a pivotal role in fluid and electrolyte movements across ducts and tubes lined by epithelia.
Cai, Zhiwei, Cami-Kobeci, Gerta, Husbands, Stephen, Khuituan, Pissared, Li, Hongyu, Liu, Jia, Sheppard, David, Wang, Yiting   +7 more
core   +2 more sources

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, Inge Stegeman, Gitte Berkers, Joost Bittermann, Ivonne Ligtenberg-van der Drift, Peter-Jan van Kipshagen, Karin de Winter-de Groot, Lucienne Speleman   +7 more
doaj   +1 more source