Results 41 to 50 of about 9,731 (210)
G970R‐CFTR Mutation (c.2908G>C) Results Predominantly in a Splicing Defect
Clinical and Translational Science, 2021 In previous work, participants with a G970R mutation in cystic fibrosis transmembrane conductance regulator (CFTR) (c.2908G>C) had numerically lower sweat chloride responses during ivacaftor treatment than participants with other CFTR gating mutations ...
Meredith C. Fidler +11 more
doaj +1 more source
Elexacaftor-Tezacaftor-Ivacaftor and Ivacaftor (Trikafta)
Canadian Journal of Health Technologies, 2021 CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments ...
openaire +3 more sources
International Forum of Allergy &Rhinology, EarlyView.ABSTRACT Background Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are highly prevalent among people with cystic fibrosis (PwCF) and negatively impact quality of life. The 40‐item Smell Identification Test (SIT) is widely used to assess psychophysical olfaction, but a CF‐specific minimal clinically important difference (MCID) has not been ...
Eugene Oh +34 more
wiley +1 more source
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 +7 more
doaj +1 more source
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 +5 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 +7 more
doaj +1 more source
Structure of ABCB1/P-Glycoprotein in the Presence of the CFTR Potentiator Ivacaftor
Membranes, 2021 ABCB1/P-glycoprotein is an ATP binding cassette transporter that is involved in the clearance of xenobiotics, and it affects the disposition of many drugs in the body.
Alessandro Barbieri +5 more
doaj +1 more source
British Journal of Clinical Pharmacology, EarlyView.Aim Elexacaftor/tezacaftor/ivacaftor (ETI) has markedly improved cystic fibrosis (CF) outcomes. However, its long‐term impact on nutrition, metabolism and liver health remains underexplored. We assessed 30‐month changes in pulmonary, nutritional, metabolic and inflammatory markers in people with CF (PwCF) homozygous for F508del.
Nicola Perrotta +5 more
wiley +1 more source
Health Technology Assessment, 2014 Background: Ivacaftor (Kalydeco®, Vertex Pharmaceuticals) is the first of a new class of drugs that target the underlying protein defect in cystic fibrosis (CF).
Penny Whiting +9 more
doaj +1 more source
Advances in bronchiectasis:endotyping, genetics, microbiome, and disease heterogeneity [PDF]
, 2018 Bronchiectasis is characterised by pathological dilation of the airways. More specifically, the radiographic demonstration of airway enlargement is the common feature of a heterogeneous set of conditions and clinical presentations.
Chalmers, James D. +2 more
core +2 more sources