The preclinical discovery and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis
Sign Up to like & getrecommendations! Published in 2020 at "Expert Opinion on Drug Discovery"
DOI: 10.1080/17460441.2020.1750592
Abstract: ABSTRACT Introduction Cystic Fibrosis (CF) is caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The most common mutation, F508del, induces protein misprocessing and loss of CFTR function. The discovery through in… read more here.
Keywords: ivacaftor; cystic fibrosis; tezacaftor; cftr ... See more keywords
Tezacaftor and ivacaftor for the treatment of cystic fibrosis
Sign Up to like & getrecommendations! Published in 2019 at "Expert Review of Respiratory Medicine"
DOI: 10.1080/17476348.2020.1682998
Abstract: ABSTRACT Introduction: Cystic fibrosis (CF) is a complex, multi-system, genetic disease affecting over 70,000 people worldwide. The underlying defect is a mutation in the CFTR gene. Dysfunctional CFTR protein results in abnormal anion movement across… read more here.
Keywords: ivacaftor; cystic fibrosis; tezacaftor; cftr ... See more keywords
Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2 Through 5 Years with Cystic Fibrosis and at Least One F508del Allele.
Sign Up to like & getrecommendations! Published in 2023 at "American journal of respiratory and critical care medicine"
DOI: 10.1164/rccm.202301-0084oc
Abstract: RATIONALE Elexacaftor/tezacaftor/ivacaftor has been shown to be safe and effective in people with cystic fibrosis (CF) aged ≥6 years with ≥1 F508del-CFTR allele but has not been studied in younger children. OBJECTIVES To evaluate the… read more here.
Keywords: ivacaftor; elexacaftor tezacaftor; tezacaftor ivacaftor; aged years ... See more keywords
Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination
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DOI: 10.1172/jci.insight.139983
Abstract: Based on its clinical benefits, Trikafta — the combination of folding correctors VX-661 (tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor) — was FDA approved for treatment of patients with cystic fibrosis (CF) carrying… read more here.
Keywords: ivacaftor; combination; tezacaftor; cftr ... See more keywords
Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis
Sign Up to like & getrecommendations! Published in 2023 at "Pharmaceuticals"
DOI: 10.3390/ph16030410
Abstract: Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly modified… read more here.
Keywords: modulator drugs; cftr; ivacaftor; cftr modulator ... See more keywords