The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches
SY Graeber, MA Mall - The Lancet, 2023 - thelancet.com
With the 2019 breakthrough in the development of highly effective modulator therapy
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …
A review of cystic fibrosis: Basic and clinical aspects
Q Chen, Y Shen, J Zheng - Animal models and experimental …, 2021 - Wiley Online Library
Cystic fibrosis is an autosomal recessive disease caused by mutations of the gene encoding
the cystic fibrosis transmembrane conductance regulator (CFTR). Here we summarize, at the …
the cystic fibrosis transmembrane conductance regulator (CFTR). Here we summarize, at the …
Rapid improvement after starting elexacaftor–tezacaftor–ivacaftor in patients with cystic fibrosis and advanced pulmonary disease
PR Burgel, I Durieu, R Chiron, S Ramel… - American journal of …, 2021 - atsjournals.org
Rationale: Elexacaftor–tezacaftor–ivacaftor is a CFTR (cystic fibrosis [CF] transmembrane
conductance regulator) modulator combination, developed for patients with CF with at least …
conductance regulator) modulator combination, developed for patients with CF with at least …
Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two F508del Alleles
SY Graeber, C Vitzthum, ST Pallenberg… - American journal of …, 2022 - atsjournals.org
Rationale: The CFTR (cystic fibrosis transmembrane conductance regulator) modulator
combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical …
combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical …
Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on sputum viscoelastic properties, airway infection and inflammation in patients with cystic fibrosis
L Schaupp, A Addante, M Völler… - European …, 2023 - Eur Respiratory Soc
Background Recent studies demonstrated that the triple combination cystic fibrosis
transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor …
transmembrane conductance regulator (CFTR) modulator therapy elexacaftor/tezacaftor …
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] Cystic fibrosis modulator therapies
S Jia, JL Taylor-Cousar - Annual review of medicine, 2023 - annualreviews.org
Cystic fibrosis (CF) is an inherited multisystemic disease that can cause progressive
bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction …
bronchiectasis, pancreatic endocrine and exocrine insufficiency, distal intestinal obstruction …
[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 …
[HTML][HTML] Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment
CA Shaughnessy, PL Zeitlin, PE Bratcher - Scientific reports, 2021 - nature.com
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR), which lead to early death due to progressive lung disease. The …
regulator (CFTR), which lead to early death due to progressive lung disease. The …
[HTML][HTML] A new era for people with cystic fibrosis
MC Bierlaagh, D Muilwijk, JM Beekman… - European Journal of …, 2021 - Springer
Cystic fibrosis is the most prevalent inherited disease caused by a defect in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene. The impaired electrolyte homeostasis …
transmembrane conductance regulator (CFTR) gene. The impaired electrolyte homeostasis …