Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer [PDF]
Therapeutic Advances in Medical OncologyBackground: Drug repurposing has emerged as an effective strategy to accelerate drug discovery. Using the pipeline established from a large collaborative drug repurposing project focused on high-grade serous ovarian cancer (HGSOC), we identified ...
Dongli Liu +17 more
doaj +2 more sources
Impact of COVID‐19 and CFTR Modulators on Cystic Fibrosis: A Real‐World Analysis of Care Patterns [PDF]
Pediatric Pulmonology, Volume 61, Issue 4, April 2026.ABSTRACT Introduction Novel therapeutics and rapid expansion of telehealth have reshaped cystic fibrosis (CF) care; however, the impact on visit patterns and equitable access across the CF population remains unclear. We characterized changes in visit patterns from 2017 to 2022 and the association of sociodemographic and clinical factors with visit ...
Alexandra C. Hinton +3 more
wiley +2 more sources
PLoS ONE, 2017 We optically measured effects of orally available ivacaftor (Kalydeco®) on sweat rates of identified glands in 3 R117H subjects, each having a unique set of additional mutations, and compared them with 5 healthy control subjects tested contemporaneously.
Jessica E Char +5 more
doaj +1 more source
European Respiratory Society International Congress best abstract preview from the allied respiratory professionals from assembly 9 [PDF]
, 2018 The 28th European Respiratory Society (ERS) International Congress [2018] in Paris features interesting research findings within the allied respiratory professional assembly (assembly 9).
Blonshine, Jason +3 more
core +1 more source
New era of cystic fibrosis: full mutational analysis and personalized therapy [PDF]
, 2017 Despite its apparently simple genetics, cystic fibrosis (CF) is a rather complex genetic disease. A lot of variability in the steps of the path from the cystic fibrosis transmembrane conductance regulator (CFTR ) gene to the clinical manifestations ...
Lucarelli, Marco
core +1 more source
Assembly and functional analysis of an S/MAR based episome with the cystic fibrosis transmembrane conductance regulator gene [PDF]
, 2018 Improving the efficacy of gene therapy vectors is still an important goal toward the development of safe and efficient gene therapy treatments. S/MAR (scaffold/matrix attached region)-based vectors are maintained extra-chromosomally in numerous cell ...
Ascenzioni, Fiorentina +10 more
core +3 more sources
Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor
Clinical and Translational Science, 2019 Drug–drug interaction (DDI) studies are described for tezacaftor/ivacaftor, a new cystic fibrosis transmembrane conductance regulator modulator therapy for the treatment of cystic fibrosis.
Varun Garg +11 more
doaj +1 more source
The potentially beneficial central nervous system activity profile of ivacaftor and its metabolites
ERJ Open Research, 2018 Ivacaftor–lumacaftor and ivacaftor are two new breakthrough cystic fibrosis transmembrane conductance modulators. The interactions of ivacaftor and its two metabolites hydroxymethylivacaftor (iva-M1) and ivacaftorcarboxylate (iva-M6) with ...
Elena K. Schneider +10 more
doaj +1 more source
Genetics and Molecular Biology, 2021 This is a descriptive cross-sectional study that aims to determine the distribution of the CFTR causing variant in a group of patients at a cystic fibrosis (CF) center in southern Brazil, as well as to describe causing variants that are treatable with ...
Eliandra da Silveira Lima +3 more
doaj +1 more source
Strategies against nonsense: oxadiazoles as translational readthrough-inducing drugs (TRIDs) [PDF]
, 2019 This review focuses on the use of oxadiazoles as translational readthrough-inducing drugs (TRIDs) to rescue the functional full-length protein expression in mendelian genetic diseases caused by nonsense mutations.
Campofelice A. +6 more
core +1 more source