|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Changes in Airway Microbiome and Inflammation with Ivacaftor Treatment in Patients with Cystic Fibrosis and the G551D Mutation.
|
31604026 |
2020 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Additive improvement in G551D CFTR-mediated Cl<sup>-</sup> secretion suggests that resveratrol could enhance ivacaftor therapy in these patients and improve CF-related rhinosinusitis.
|
30152192 |
2019 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Ivacaftor improves QOL in the R, P, and S domains in G551D CF patients, although QOL instruments validated for CRS may not translate well to CF CRS patients because symptom burden was surprisingly low.
|
30472785 |
2019 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
We report the results of an observational study investigating its effects in CF patients with non-G551D gating mutations.
|
29685811 |
2019 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Combined with the concentration-dependent decrease in sweat chloride concentration, results show that GLPG1837 increases CFTR activity in G551D-CF patients.
|
31147302 |
2019 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Although ivacaftor (VX-770) alone and ivacaftor in combination with lumacaftor (VX-809) improve lung function in CF patients with the Gly551Asp and del508Phe mutations, respectively, the effects of these drugs on the function of human CF macrophages are unknown.
|
29146575 |
2018 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
In people with CF bearing G551D or R117H mutations, ivacaftor did not change the average ASL pH; however reductions in sweat Cl- concentration correlated with elevations of ASL pH.
|
30089726 |
2018 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Ivacaftor produces significant clinical benefit in patients with cystic fibrosis (CF) with the G551D mutation.
|
29037527 |
2018 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
For adults and children aged 6 years and older with CF due to gating mutations other than G551D or R117H, the guideline panel made a conditional recommendation for treatment with IVA.
|
29342367 |
2018 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Retrospective observational study of French patients with cystic fibrosis and a Gly551Asp-CFTR mutation after 1 and 2years of treatment with ivacaftor in a real-world setting.
|
28711222 |
2018 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor.
|
28651844 |
2018 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiator ivacaftor (Kalydeco®) improves clinical outcome in G551D cystic fibrosis (CF) patients.
|
28445004 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Changes of CFTR functional measurements and clinical improvements in cystic fibrosis patients with non p.Gly551Asp gating mutations treated with ivacaftor.
|
27659740 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Variability of sweat chloride concentration in subjects with cystic fibrosis and G551D mutations.
|
26996268 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Previous work indicates that ivacaftor improves cystic fibrosis transmembrane conductance regulator (CFTR) activity and lung function in people with cystic fibrosis and G551D-CFTR mutations but does not reduce density of bacteria or markers of inflammation in the airway.
|
28222269 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
The potentiator ivacaftor (VX-770) is approved for treating CF patients with G551D and other gating mutations.
|
27707539 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
This study analyzed plasma fatty acid levels and urine prostaglandin E metabolites (PGE-M) in 40 subjects with CF participating in the G551D observational (GOAL) study who demonstrated response to the medication by a significant decrease in sweat Cl levels.
|
27473897 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
This pilot study evaluated the effect of short- and long-term ivacaftor treatment on hyperpolarized <sup>3</sup>He-magnetic resonance imaging (MRI)-defined ventilation defects in patients with cystic fibrosis aged ≥12years with a G551D-CFTR mutation.
|
28132845 |
2017 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
In this study, we provide the first evidence that ivacaftor improves BMD in CF patients carrying the p.Gly551Asp mutation.
|
27745802 |
2016 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Alterations in blood leukocytes of G551D-bearing cystic fibrosis patients undergoing treatment with ivacaftor.
|
25769931 |
2016 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
The potentiator VX-770 (ivacaftor/KALYDECO™) targets defective gating of CFTR and has been approved for treatment of cystic fibrosis (CF) subjects carrying G551D, S1251N or one of 8 other mutations.
|
27160424 |
2016 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: Safety and efficacy in an expanded access program in the United States.
|
25682022 |
2016 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
The most common mutations in CFTR are a deletion of a phenylalanine residue at position 508 (ΔF508-CFTR, 70-80 % of CF phenotypes) and a Gly551Asp substitution (G551D-CFTR, 4-5 % of alleles), which lead to decreased or almost abolished Cl(-) channel function, respectively.
|
26874684 |
2016 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
The potentiator VX-770 was the first CFTR modulator approved by the FDA for treatment of CF patients with the gating mutation G551D.
|
27402691 |
2016 |
|
Cystic Fibrosis
|
|
0.900 |
GeneticVariation
|
BEFREE |
After several successful clinical trials the potentiator, ivacaftor, is now licenced for use in adults and children (>six years), with CF bearing the class III G551D mutation and FDA licence was recently expanded to include 8 additional class III mutations.
|
24932877 |
2015 |