The future of cystic fibrosis care: a global perspective
Executive summary The past six decades have seen remarkable improvements in health
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …
outcomes for people with cystic fibrosis, which was once a fatal disease of infants and young …
CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine
M Lopes-Pacheco - Frontiers in pharmacology, 2020 - frontiersin.org
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …
A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele
ET Zemanick, JL Taylor-Cousar, J Davies… - American journal of …, 2021 - atsjournals.org
Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and
safe in patients≥ 12 years of age with cystic fibrosis and at least one F508del-CFTR (cystic …
safe in patients≥ 12 years of age with cystic fibrosis and at least one F508del-CFTR (cystic …
Cystic fibrosis: a review
T Ong, BW Ramsey - Jama, 2023 - jamanetwork.com
Importance Cystic fibrosis, a genetic disorder defined by variants in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …
transmembrane conductance regulator (CFTR) gene, affects more than 30 000 individuals in …
[HTML][HTML] Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del
JL Taylor-Cousar, A Munck, EF McKone… - New england journal …, 2017 - Mass Medical Soc
Background Combination treatment with the cystic fibrosis transmembrane conductance
regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to …
regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to …
[HTML][HTML] Tezacaftor–ivacaftor in residual-function heterozygotes with cystic fibrosis
SM Rowe, C Daines, FC Ringshausen… - … England Journal of …, 2017 - Mass Medical Soc
Background Cystic fibrosis is an autosomal recessive disease caused by mutations in the
CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show …
CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show …
Effects of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in patients with cystic fibrosis and one or two F508del …
SY Graeber, DM Renz, M Stahl… - American journal of …, 2022 - atsjournals.org
Rationale: We recently demonstrated that triple-combination CFTR (cystic fibrosis
transmembrane conductance regulator) modulator therapy with elexacaftor/tezacaftor …
transmembrane conductance regulator) modulator therapy with elexacaftor/tezacaftor …
[HTML][HTML] CFTR modulator theratyping: Current status, gaps and future directions
JP Clancy, CU Cotton, SH Donaldson… - Journal of Cystic …, 2019 - Elsevier
Background New drugs that improve the function of the cystic fibrosis transmembrane
conductance regulator (CFTR) protein with discreet disease-causing variants have been …
conductance regulator (CFTR) protein with discreet disease-causing variants have been …
Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype
ME McGarry, SA McColley - Pediatric pulmonology, 2021 - Wiley Online Library
Background Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are
disease‐modifying medications for cystic fibrosis (CF) and are shown to be efficacious for …
disease‐modifying medications for cystic fibrosis (CF) and are shown to be efficacious for …
Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function …
Rationale: The triple-combination regimen elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)
was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis …
was shown to be safe and efficacious in children aged 6 through 11 years with cystic fibrosis …