Modulators of CFTR. Updates on clinical development and future directions.
Eur J Med Chem
; 213: 113195, 2021 Mar 05.
Article
em En
| MEDLINE
| ID: mdl-33524685
ABSTRACT
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.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Quinolonas
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Regulador de Condutância Transmembrana em Fibrose Cística
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Fibrose Cística
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Benzodioxóis
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Desenvolvimento de Medicamentos
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Aminofenóis
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Aminopiridinas
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Indóis
Tipo de estudo:
Diagnostic_studies
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Prognostic_studies
Limite:
Humans
Idioma:
En
Revista:
Eur J Med Chem
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
França