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Charlotte M. McKee
Researcher at Vertex Pharmaceuticals
Publications - 11
Citations - 4410
Charlotte M. McKee is an academic researcher from Vertex Pharmaceuticals. The author has contributed to research in topics: Ivacaftor & Lumacaftor. The author has an hindex of 9, co-authored 11 publications receiving 2815 citations.
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Journal ArticleDOI
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Jennifer L. Taylor-Cousar,Anne Munck,Edward F. McKone,Cornelis K. van der Ent,Alexander Moeller,Christopher Simard,Linda T Wang,Edward P. Ingenito,Charlotte M. McKee,Yimeng Lu,J Lekstrom-Himes,J. Stuart Elborn +11 more
TL;DR: These data show that lumacaftor in combination with ivacaftors provided a benefit for patients with cystic fibrosis homozygous for the Phe508del CFTR mutation.
Journal ArticleDOI
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
Peter G. Middleton,Marcus A. Mall,Pavel Dřevínek,Larry C. Lands,Edward F. McKone,Deepika Polineni,Bonnie W. Ramsey,Jennifer L. Taylor-Cousar,Elizabeth Tullis,François Vermeulen,Gautham Marigowda,Charlotte M. McKee,Samuel M. Moskowitz,Nitin Nair,Jessica H. Savage,Christopher Simard,S. Tian,David Waltz,Fengjuan Xuan,Steven M. Rowe,Raksha Jain +20 more
TL;DR: Elexacaftor-tezacaft or-ivacaft or was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective.
Journal ArticleDOI
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation : a double-blind, randomised, phase 3 trial
Harry G.M. Heijerman,Edward F. McKone,Damian G. Downey,Eva Van Braeckel,Steven M. Rowe,Elizabeth Tullis,Marcus A. Mall,John Welter,Bonnie W. Ramsey,Charlotte M. McKee,Gautham Marigowda,Samuel M. Moskowitz,David Waltz,Patrick R Sosnay,Christopher Simard,Neil Ahluwalia,Fengjuan Xuan,Yaohua Zhang,Jennifer L. Taylor-Cousar,Karen McCoy +19 more
TL;DR: Elexacaftor plus tezacaftorplus ivacaftors provided clinically robust benefit compared with tezacftor plus ivACaftor alone, with a favourable safety profile, and shows the potential to lead to transformative improvements in the lives of people with cystic fibrosis who are homozygous for the F508del mutation.
Journal ArticleDOI
VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
Dominic Keating,Gautham Marigowda,Lucy D. Burr,Cori L. Daines,Marcus A. Mall,Edward F. McKone,Bonnie W. Ramsey,Steven M. Rowe,Laura A. Sass,Elizabeth Tullis,Charlotte M. McKee,Samuel M. Moskowitz,Sarah Robertson,Jessica H. Savage,Christopher Simard,Fredrick Van Goor,David Waltz,Fengjuan Xuan,Young Tomothy John,Jennifer L. Taylor-Cousar +19 more
TL;DR: The use of VX‐445–tezacaftor–ivacaftor to target Phe508del CFTR protein resulted in increased CFTR function in vitro and translated to improvements in patients with cystic fibrosis with one or two Phe48del alleles.
Journal ArticleDOI
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis
Steven M. Rowe,Cori L. Daines,Felix C. Ringshausen,Eitan Kerem,John W Wilson,Elizabeth Tullis,Nitin Nair,Christopher Simard,Linda Han,Edward P. Ingenito,Charlotte M. McKee,J Lekstrom-Himes,Jane C. Davies +12 more
TL;DR: CFTR modulator therapy with tezacaftor–ivacaftor or ivacaftors alone was efficacious in patients with cystic fibrosis who were heterozygous for the Phe508del deletion and a CFTR residual‐function mutation.