Cystic fibrosis: a disease in transformation, yet more work to be done!
BW Ramsey, SC Bell - American Journal of Respiratory and Critical …, 2022 - atsjournals.org
What have we learned from real-world experience about highly effective CFTR modulation
and its impact on the lives of patients with cystic fibrosis? In 2019, the triple combination …
and its impact on the lives of patients with cystic fibrosis? In 2019, the triple combination …
Global access to affordable CFTR modulator drugs: Time for action!
The rapid advancements in cystic fibrosis (CF) treatment in the last two decades and
improvement in CF outcomes is surely one of the greatest achievements in modern …
improvement in CF outcomes is surely one of the greatest achievements in modern …
Evaluation of elexacaftor–tezacaftor–ivacaftor treatment in individuals with cystic fibrosis and CFTRN1303K in the USA: a prospective, multicentre, open-label, single …
GM Solomon, RW Linnemann, R Rich… - The Lancet …, 2024 - thelancet.com
Background CFTR modulators are approved for approximately 90% of people with cystic
fibrosis in the USA and provide substantial clinical benefit. N1303K (Asn1303Lys), one of …
fibrosis in the USA and provide substantial clinical benefit. N1303K (Asn1303Lys), one of …
[引用][C] A contemporary assessment of CFTR modulator use and eligibility
JL Rubin, JL Kreindler - Journal of Cystic Fibrosis, 2024 - cysticfibrosisjournal.com
The treatment of cystic fibrosis (CF) changed dramatically with the advent of cystic fibrosis
transmembrane conductance regulator (CFTR) modulator therapies that address the …
transmembrane conductance regulator (CFTR) modulator therapies that address the …
Sustained Improvement of Cystic Fibrosis Lung Disease following Initiation of Elexacaftor/Tezacaftor/Ivacaftor Therapy: Lessons from Real-World Studies
E Steinke, M Stahl - American Journal of Respiratory and Critical …, 2023 - atsjournals.org
The last four decades have seen significant advances in the therapy of cystic fibrosis (CF)
with the recent advent of cystic fibrosis transmembrane conductance regulator (CFTR) …
with the recent advent of cystic fibrosis transmembrane conductance regulator (CFTR) …
Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del …
Background Tezacaftor–ivacaftor is an approved cystic fibrosis transmembrane conductance
regulator (CFTR) modulator shown to be efficacious and generally safe and well tolerated …
regulator (CFTR) modulator shown to be efficacious and generally safe and well tolerated …
Impact of the expanded label for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with no F508del variant in the USA
EA Cromwell, JS Ostrenga… - European …, 2024 - publications.ersnet.org
Background Elexacaftor/tezacaftor/ivacaftor (ETI), which is approved for people with cystic
fibrosis (pwCF) with a F508del variant, was further approved based on in vitro data in the …
fibrosis (pwCF) with a F508del variant, was further approved based on in vitro data in the …
[HTML][HTML] Bridging the gap: Challenging lung infections and clinical trial development in cystic fibrosis
DG Downey, NJ Simmonds… - Journal of Cystic …, 2024 - Elsevier
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy has
transformed the lives of many people with CF (pwCF) with improved quality of life (QoL) …
transformed the lives of many people with CF (pwCF) with improved quality of life (QoL) …
Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy
Cystic fibrosis is a multiorgan disease caused by impaired function of the cystic fibrosis
transmembrane conductance regulator (CFTR). Since the introduction of the CFTR …
transmembrane conductance regulator (CFTR). Since the introduction of the CFTR …
[HTML][HTML] Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: interim results of a long-term registry-based study
JK Bower, N Volkova, N Ahluwalia, G Sahota… - Journal of Cystic …, 2023 - Elsevier
Background Phase 3 clinical trials showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA)
was safe and efficacious in people with cystic fibrosis (CF) with≥ 1 F508del-CFTR allele. To …
was safe and efficacious in people with cystic fibrosis (CF) with≥ 1 F508del-CFTR allele. To …