Results 41 to 50 of about 7,376 (199)
, 2022 Background and purpose: Cystic fibrosis (CF) is associated with a myriad of respiratory complications including increased susceptibility to lung infections and inflammation. Progressive inflammatory insults lead to airway damage and remodeling, resulting
Andrew Jarnicki (322440) +3 more
core +2 more sources
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
Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review
, 2021 Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF.
Keval Chandarana +6 more
core +1 more source
Regulatory harmonization: Evolution, globalization and future directions
British Journal of Clinical Pharmacology, EarlyView.Regulatory harmonization has become an increasingly important and accepted approach to streamline regulatory review processes and expedite access to safe, effective and high‐quality medicines globally. This review explores the evolution and current status of regulatory harmonization, convergence and reliance initiatives.
Orin Chisholm +2 more
wiley +1 more source
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 +28 more
core +2 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 +7 more
doaj +1 more source
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 +12 more
wiley +1 more source
Comparing the Effectiveness of Ivacaftor and GLPG1837 [PDF]
, 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 +4 more
core +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
The use of targeted therapy lumacaftor/ivacaftor in patients with cystic fibrosis
Медицинский совет, 2022 Accepted, basic therapy of cystic fibrosis (CF), until recently, was symptomatic and aimed at slowing down pathological processes, mainly from the respiratory system and gastrointestinal tract, caused by a defect in the CFTR gene.
A. G. Chermensky +3 more
doaj +1 more source