Révolution dans le traitement de la mucoviscidose [Revolution in the treatment of cystic fibrosis]

Détails

Ressource 1Télécharger: RMS_698_1229.pdf (2899.14 [Ko])
Etat: Public
Version: Final published version
Licence: CC BY-NC-ND 4.0
ID Serval
serval:BIB_EC0F290D1773
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Institution
Titre
Révolution dans le traitement de la mucoviscidose [Revolution in the treatment of cystic fibrosis]
Périodique
Revue medicale suisse
Auteur⸱e⸱s
Sauty A., Plojoux J., Mornand A., Blanchon S., Koutsokera A.
ISSN
1660-9379 (Print)
ISSN-L
1660-9379
Statut éditorial
Publié
Date de publication
17/06/2020
Peer-reviewed
Oui
Volume
16
Numéro
698
Pages
1229-1235
Langue
français
Notes
Publication types: Journal Article ; Review
Publication Status: ppublish
Résumé
Cystic Fibrosis is a genetic disorder resulting in the absence or dysfunction of the CFTR protein, a chloride channel present on the surface of epithelia, particularly respiratory. Until recently, treatments only concerned the consequences of the disease. But a new type of molecules called « modulators », is already available to some patients and targets the origin of the disease. « Modulators » are divided into « potentiators », which improve the transport of chloride by the CFTR protein, and « correctors », increasing the amount of CFTR proteins. An oral triple therapy combining a potentiator and two correctors has just been approved in the USA and will treat 85 % of patients. The clinical benefit of « modulators » is remarkable, and these drugs are revolutionizing the treatment of Cystic Fibrosis.
Mots-clé
Cystic Fibrosis/metabolism, Cystic Fibrosis/therapy, Cystic Fibrosis Transmembrane Conductance Regulator/genetics, Cystic Fibrosis Transmembrane Conductance Regulator/metabolism, Humans, Mutation
Pubmed
Création de la notice
06/07/2020 13:45
Dernière modification de la notice
20/08/2022 7:15
Données d'usage