Results 51 to 60 of about 12,467 (245)
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
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
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
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
Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants. [PDF]
, 2019 Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the
Finkbeiner, Walter E +7 more
core
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. +18 more
core +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
Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation
Anales de Pediatría (English Edition), 2019 Introduction: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has been shown to improve the nutritional status and lung function of cystic fibrosis patients with the G551D mutation in clinical trials.
David Gomez-Pastrana +5 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
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