Results 41 to 50 of about 6,532 (197)

Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del [PDF]

, 2017
Background Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis.Methods In ...
Anne Munck, McKee, Charlotte, Ingenito, Edward P., Moeller, Alexander, van der Ent, Cornelis K, Ingenito, Edward P, Munck, Anne, J. Stuart Elborn, Edward P. Ingenito, McKone, Edward F., Simard, Christopher, Wang, Linda T., Edward F. McKone, Julie Lekstrom-Himes, Christopher Simard, Charlotte McKee, Jennifer L. Taylor-Cousar, Elborn, J Stuart, Yimeng Lu, Lekstrom-Himes, Julie, Cornelis K. van der Ent, Alexander Moeller, Taylor-Cousar, Jennifer L, Taylor-Cousar, Jennifer L., Linda T. Wang, Lu, Yimeng, McKone, Edward F, Van Der Ent, Cornelis K., Wang, Linda T   +28 more
core   +1 more source

Combined CFTR modulator therapies are linked with anabolic benefits and insulin-sparing in cystic fibrosis-related diabetes

Journal of Clinical & Translational Endocrinology, 2023
Aims: Combined CFTR modulator therapies have dramatically altered pulmonary outcomes in patients with cystic fibrosis (CF). Their impact on glucose metabolism requires further investigations.
Fabian Lurquin, Sophie Gohy, Michel P. Hermans, Vanessa Preumont   +3 more
doaj   +1 more source

Optimization of pharmaceutical research and development by early‐phase assessment of investigational medicinal products

British Journal of Clinical Pharmacology, EarlyView.
Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló, Attila Imre, Gábor Varga, Sándor Kovács, György Tímár, Balázs Nagy   +5 more
wiley   +1 more source

Antibacterial properties of the CFTR potentiator ivacaftor

, 2014
BackgroundIvacaftor increases CFTR channel activity and improves pulmonary function for individuals bearing a G551D mutation. Because ivacaftor structurally resembles quinolone antibiotics, we tested the hypothesis that ivacaftor possesses antibacterial ...
Gansemer, Nick D., Abou Alaiwa, Mahmoud H., Dohrn, Cassie L., Welsh, Michael J., Diekema, Daniel J., Reznikov, Leah R., Stoltz, David A.   +6 more
core   +1 more source

Alteration of Membrane Cholesterol Content Plays a Key Role in Regulation of Cystic Fibrosis Transmembrane Conductance Regulator Channel Activity

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, Kirsten A. Cottrill, Kerry M. Strickland, Sarah A. Mashburn, Michael Koval, Nael A. McCarty   +5 more
doaj   +1 more source

Pregnancy‐related effect on elexacaftor, tezacaftor and ivacaftor pharmacokinetics in women with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Aim The number of pregnancies among women with cystic fibrosis (wwCF) has steadily increased over the past decade. However, the pharmacokinetics (PK) of elexacaftor–tezacaftor–ivacaftor (ETI) during gestation remains uncharacterized, despite its widespread use in this population.
Paulette Magnas, Naim Bouazza, Frantz Foissac, Saïk Urien, Léo Froelicher‐Bournaud, Gabrielle Lui, Victoria Guillemart, Nicolas Carlier, Jennifer Da Silva, Johanna Fesenbeckh, Reem Kanaan, Isabelle Honoré, Clémence Martin, Jean‐Marc Treluyer, Vassilis Tsatsaris, Pierre‐Régis Burgel, Sihem Benaboud   +16 more
wiley   +1 more source

SLC26A9 Gene Is Associated With Lung Function Response to Ivacaftor in Patients With Cystic Fibrosis

Frontiers in Pharmacology, 2018
Ivacaftor is a drug used to treat cystic fibrosis (CF) patients carrying specific gating CFTR mutations. Interpatient variability in the lung response has been shown to be partly explained by rs7512462 in the Solute Carrier Family 26 Member 9 (SLC26A9 ...
Harriet Corvol, Harriet Corvol, Julie Mésinèle, Isman-Hassan Douksieh, Isman-Hassan Douksieh, Lisa J. Strug, Pierre-Yves Boëlle, Loïc Guillot   +7 more
doaj   +1 more source

Impact of cystic fibrosis transmembrane conductance regulator modulator therapies on liver stiffness and liver enzymes: An observational perspective single‐center cohort study

JPGN Reports, EarlyView.
Abstract Objectives The efficacy of cystic fibrosis transmembrane conductance regulator (CFTR)‐modulator therapies in preventing or ameliorating cystic fibrosis liver disease (CFLD) by correcting CFTR in cholangiocytes is not well‐documented. This study aimed to assess liver function during CFTR‐modulators.
Laura Giugliano, Elisabetta Augustoni, Antonio Pizzol, Anna Opramolla, Cristina Chiadò, Anna Zagaria, Irene Esposito, Giulia Roberto, Alice Ponte, Andrea Evangelista, Paola Quarello, Michele Pinon, Pier Luigi Calvo   +12 more
wiley   +1 more source

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles [PDF]

, 2018
BACKGROUND The next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659–tezacaftor–ivacaftor), was developed to restore the function of Phe508del CFTR protein
Moskowitz, Samuel M., Mall, Marcus A., Tullis, Elizabeth, Horsley, Alexander; id_orcid, Van Goor, Fredrick, Young, Tim, Uluer, Ahmet, Brown, Cynthia, Mckee, Charlotte M., Robertson, Sarah, Davies, Jane C., Mckone, Edward F., Ramsey, Bonnie W., Plant, Barry J., Simard, Christopher, Waltz, David, Xuan, Fengjuan, Horsley, Alexander, Taylor-cousar, Jennifer L., Shilling, Rebecca A., Prais, Dario, Rowe, Steven M.   +21 more
core   +3 more sources

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Biomedical Papers, 2016
Aims: Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ivacaftor who were followed for up to 6 years together ...
Libor Fila, Lucie Valentova Bartakova, Alzbeta Grandcourtova, Miloslav Marel, Radovan Drnek, Alena Bilkova, Milan Macek, Pavel Drevinek   +7 more
doaj   +1 more source