Results 81 to 90 of about 6,532 (197)

Ivacaftor ameliorates mucus burden, bacterial load, and inflammation in acute but not chronic P. aeruginosa infection in hG551D rats

Respiratory Research
Background Newly approved highly effective modulation therapies (HEMT) have been life-changing for people with CF. Although these drugs have resulted in significant improvements in lung function and exacerbation rate, bacterial populations in the lung ...
Johnathan D. Keith, Mikayla Murphree-Terry, Gretchen Bollar, Ashley M. Oden, Ian H. Doty, Susan E. Birket   +5 more
doaj   +1 more source

M- and C-sweat secretion (±) ivacaftor for CF subject 1: male, F508del/G551D.

, 2014
(A) Larger M-sweat responses (+) ivacaftor were seen (uniquely) in this subject. Scatter plot shows gland-by-gland volumes measured 7.5 min post MCh injection before any appreciable mergers for 46 glands.
Jessica E. Char (476386), Hyung-ju Cho (5656717), Ewart A. C. Thomas (476393), Colleen Dunn (476388), Il-Ho Park (476392), Richard B. Moss (521193), Jeffrey J. Wine (207605), Carlos Milla (476390), Zoe Davies (521192), Eric Frisbee (476389), Marlene H. Wolfe (476396), Jin Hyeok Jeong (521191)   +11 more
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Ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation [PDF]

European Respiratory Review, 2013
Cystic fibrosis (CF) is an autosomal recessive lethal disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes for CFTR, an epithelial cell-surface expressed protein responsible for the transport of ...
Isabelle Sermet-Gaudelus
doaj  

Effects of ivacaftor on severely ill patients with cystic fibrosis carrying a G551D mutation

, 2013
BackgroundRecently, ivacaftor, a CFTR-potentiator, has been shown to be effective and safe in patients with cystic fibrosis carrying a G551D mutation and moderately impaired lung function.
Helge Hebestreit, Hebestreit, Helge, Käding, Manfred, Sauer-Heilborn, Annette, Mainz, Jochen G., Manfred Käding, Annette Sauer-Heilborn, Jochen G. Mainz, Rainald Fischer, Fischer, Rainald   +9 more
core   +1 more source

Case report of an unexpected reaction associated with Lumacaftor/ivacaftor therapy for cystic fibrosis

, 2019
Lumacaftor/ivacaftor increases cystic fibrosis transmembrane conductance regulator (CFTR) activity and is an effective treatment for cystic fibrosis (CF) patients that are homozygous for the F508del mutation.
Farrell C (8101806)
core   +1 more source

Impact of the CFTR-potentiator ivacaftor on airway microbiota in cystic fibrosis patients carrying a G551D mutation.

PLoS ONE, 2015
BackgroundAirway microbiota composition has been clearly correlated with many pulmonary diseases, and notably with cystic fibrosis (CF), an autosomal genetic disorder caused by mutation in the CF transmembrane conductance regulator (CFTR).
Cédric Bernarde, Marlène Keravec, Jérôme Mounier, Stéphanie Gouriou, Gilles Rault, Claude Férec, Georges Barbier, Geneviève Héry-Arnaud   +7 more
doaj   +1 more source

Real-world population pharmacokinetics of tezacaftor-ivacaftor in children with cystic fibrosis:The SYM-CF study [PDF]

Aims: The clinical effectiveness of tezacaftor-ivacaftor in children with cystic fibrosis (cwCF) varies; some patients respond while others do not or have adverse effects.
Vonk, Steffie E.M., Mathôt, Ron A.A., Terheggen-Lagro, Suzanne W.J., Maitland–van der Zee, Anke Hilse, Haarman, Eric G., Janssens, Hettie M., Kemper, E. Marleen   +6 more
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Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review

Frontiers in Endocrinology
IntroductionCystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CFTR gene, leading to impaired chloride transport, thickened mucus, and multiorgan dysfunction. Among its complications, cystic fibrosis-related diabetes (CFRD)
Paola Giordano, Giuseppina Leonetti, Vanja Granberg, Rosa Maria Pia Casolino, Giuseppe Lassandro, Maurizio Delvecchio, Giovanna Linguiti   +6 more
doaj   +1 more source

Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor

, 2020
Prior to the use of cystic fibrosis (CF) modulator therapy, exocrine pancreatic insufficiency in CF was thought to be irreversible. Improvement in pancreatic function on ivacaftor has been reported in open label studies in 1-5 year olds. The mechanism by
Davies, JC, Nichols, AL, Jones, D, Carr, SB   +3 more
core   +1 more source

Comparing the Effectiveness of Ivacaftor and GLPG1837

, 2021
Cystic Fibrosis is a fatal autosomal recessive genetic disease that effects more than 70,000 people worldwide. This disease is caused by a mutation within the CFTR causing an excess of mucus within the lungs, and effects other cells that produce bodily ...
Schmitt Lavin, Emily, Reyes, Saimi, Sankavaram, Vibha, Sikora, Arthur, Carreras, Jordan Nichole   +4 more
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