An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2–5 years (KLIMB)

Autor: Rosenfeld, Margaret, Cunningham, Steve, Harris, William T., Lapey, Allen, Regelmann, Warren E., Sawicki, Gregory S., Southern, Kevin W., Chilvers, Mark, Higgins, Mark, Tian, Simon, Cooke, Jon, Davies, Jane C.

Publikováno v: In Journal of Cystic Fibrosis November 2019 18(6):838-843

Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR

Autor: Horsley, Alexander, Donaldson, Scott H, Pilewski, Joseph M., Griese, Matthias, Cooke, Jon, Viswanathan, Lakshmi, Tullis, Elizabeth, Davies, Jane C., Lekstrom-Himes, Julie A., Wang, Linda T.

Publikováno v: Horsley, A, Donaldson, S H, Pilewski, J M, Griese, M, Cooke, J, Viswanathan, L, Tullis, E, Davies, J C, Lekstrom-Himes, J A & Wang, L T 2018, ' Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR ', American Journal of Respiratory and Critical Care Medicine, vol. 197, no. 2 . https://doi.org/10.1164/rccm.201704-0717OC

Rationale: Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::39abdb03542155bf2ea5f41a6f10545c
https://www.research.manchester.ac.uk/portal/en/publications/tezacaftorivacaftor-in-subjects-with-cystic-fibrosis-and-f508delf508delcftr-or-f508delg551dcftr(99940aac-7bf9-40fc-8d3b-13f50b57efee).html

Effects of Ivacaftor in CF Patients with R117H-CFTR

Autor: Moss, Richard B, Flume, Patrick A, J Stuart Elborn, Cooke, Jon, Rowe, Steven M, Mccolley, Susanna A, Rubenstein, Ronald C, Pilewski, Joseph, Higgins, Mark

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::428ee7c6302ccd193f15fd7bf4b944be

Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTRgating mutation (KIWI): an open-label, single-arm study

Autor: Davies, Jane C, Cunningham, Steve, Harris, William T, Lapey, Allen, Regelmann, Warren E, Sawicki, Gregory S, Southern, Kevin W, Robertson, Sarah, Green, Yulia, Cooke, Jon, Rosenfeld, Margaret

Publikováno v: The Lancet Respiratory Medicine; February 2016, Vol. 4 Issue: 2 p107-115, 9p

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTRmutation: a double-blind, randomised controlled trial

Autor: Moss, Richard B, Flume, Patrick A, Elborn, J Stuart, Cooke, Jon, Rowe, Steven M, McColley, Susanna A, Rubenstein, Ronald C, Higgins, Mark

Publikováno v: The Lancet Respiratory Medicine; July 2015, Vol. 3 Issue: 7 p524-533, 10p