Scientific article
Review
French

Révolution dans le traitement de la mucoviscidose

Published inRevue médicale suisse, vol. 16, no. 698, p. 1229-1235
Publication date2020
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/therapy
  • Cystic Fibrosis Transmembrane Conductance Regulator/genetics/metabolism
  • Humans
  • Mutation
Citation (ISO format)
SAUTY, Alain et al. Révolution dans le traitement de la mucoviscidose. In: Revue médicale suisse, 2020, vol. 16, n° 698, p. 1229–1235.
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Article (Published version)
accessLevelRestricted
Identifiers
Journal ISSN1660-9379
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