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

Details

Serval ID
serval:BIB_EC0F290D1773
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Révolution dans le traitement de la mucoviscidose [Revolution in the treatment of cystic fibrosis]
Journal
Revue medicale suisse
Author(s)
Sauty A., Plojoux J., Mornand A., Blanchon S., Koutsokera A.
ISSN
1660-9379 (Print)
ISSN-L
1660-9379
Publication state
Published
Issued date
17/06/2020
Peer-reviewed
Oui
Volume
16
Number
698
Pages
1229-1235
Language
french
Notes
Publication types: Journal Article ; Review
Publication Status: ppublish
Abstract
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.
Keywords
Cystic Fibrosis/metabolism, Cystic Fibrosis/therapy, Cystic Fibrosis Transmembrane Conductance Regulator/genetics, Cystic Fibrosis Transmembrane Conductance Regulator/metabolism, Humans, Mutation
Pubmed
Create date
06/07/2020 13:45
Last modification date
19/07/2020 6:26
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