[Therapeutic revolution for cystic fibrosis]

THERAPEUTIC REVOLUTION FOR CYSTIC FIBROSIS. Cystic fibrosis is an autosomal recessive disease due to mutations or variants in the CFTR gene which encodes the CFTR protein. Since the cloning of the CFTR gene thirty years ago, intense basic research paved the way to the development of therapies called CFTR modulators. The first CFTR modulator was marketed in 2012 and they are now available for more than 85% of patients in France. Patients are eligible for these oral treatments depending on the variants they bear. CFTR modulators bind to the CFTR protein, improve its function and improvement of clinical disease is observed. For the most effective CFTR modulators, respiratory symptoms decrease or disappear, respiratory function is improved, and it is likely that life expectancy will also improve. These different CFTR modulators are a therapeutic revolution for most people with cystic fibrosis.