Article
[News drugs and evolution towards personalized treatment for cystic fibrosis].
Revue medicale de Liege - 1 May 2020
Boboli H, Pirson J, Seghaye M C, Kempeneers C
Abstract excerpt
Cystic fibrosis is a genetic disorder responsible for the production of a defective transmembrane protein. In recent years, new protein modulators have been developed. They aim to treat the underlying cause of the disease. The results on the biomarkers of the function of the CFTR protein and on the clinical outcomes are very encouraging. However, there is an individual heterogeneity in the response to modulators...
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