Modulators of CFTR. Updates on clinical development and future directions.
Aminophenols
/ chemical synthesis
Aminopyridines
/ chemical synthesis
Benzodioxoles
/ chemical synthesis
Clinical Trials as Topic
Cystic Fibrosis
/ diagnosis
Cystic Fibrosis Transmembrane Conductance Regulator
/ genetics
Drug Development
Humans
Indoles
/ chemical synthesis
Quinolones
/ chemical synthesis
CFTR modulators
Corrector
Cystic fibrosis
Elexacaftor
Ivacaftor
Lumacaftor
Potentiator
Tezacafor
Journal
European journal of medicinal chemistry
ISSN: 1768-3254
Titre abrégé: Eur J Med Chem
Pays: France
ID NLM: 0420510
Informations de publication
Date de publication:
05 Mar 2021
05 Mar 2021
Historique:
received:
03
08
2020
revised:
08
01
2021
accepted:
11
01
2021
pubmed:
2
2
2021
medline:
1
5
2021
entrez:
1
2
2021
Statut:
ppublish
Résumé
Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in the Caucasian population. It is due to mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat the downstream consequences of CFTR mutations, have increased survival. Better knowledge of the CFTR protein has enabled pharmacologic therapy aiming to restore mutated CFTR expression and function. These CFTR "modulators" have revolutionised the CF therapeutic landscape, with the potential to transform prognosis for a considerable number of patients. This review provides a brief summary of their mechanism of action and presents a thorough review of the results obtained from clinical trials of CFTR modulators.
Identifiants
pubmed: 33524685
pii: S0223-5234(21)00044-1
doi: 10.1016/j.ejmech.2021.113195
pii:
doi:
Substances chimiques
Aminophenols
0
Aminopyridines
0
Benzodioxoles
0
CFTR protein, human
0
Indoles
0
Quinolones
0
tezacaftor
0
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
ivacaftor
1Y740ILL1Z
lumacaftor
EGP8L81APK
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
113195Informations de copyright
Copyright © 2021 Elsevier Masson SAS. All rights reserved.
Déclaration de conflit d'intérêts
Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.