Abstract

DE LA HOZ, Diana; VILLAMIL OSORIO, Milena  and  RESTREPO-GUALTEROS, Sonia M. Cystic fibrosis transmembrane conductance regulator modulators: Present and future in cystic fibrosis treatment. A review. Arch. argent. pediatr. [online]. 2019, vol.117, n.2, pp.e131-e136. ISSN 0325-0075.  http://dx.doi.org/10.5546/aap.2019.e131.

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are the present and future of drug management for patients with cystic fibrosis. The objective of this article is to review this therapeutic option. Scientific articles were reviewed by searching the MedLine database, which is available through the Cystic Fibrosis Foundation's official website, from 2009 to 2018, in English. Twelve articles about the current status of research in CFTR modulators were selected without restrictions regarding the type of study. To date, the United States Food and Drug Administration has approved three modulators: ivacaftor, lumacaftor + ivacaftor, and tezacaftor + ivacaftor, while other 11 drugs are being studied in different investigation phases. CFTR modulator therapy is a developing reality aimed at the highest goal of personalized medicine and promises to improve the quality of life of cystic fibrosis patients.

Keywords : Cystic fibrosis; Mutation; Therapy; Cystic fibrosis transmembrane conductance regulator.