Modulators of CFTR. Updates on clinical development and future directions

Cystic fibrosis (CF) is the most frequent life-limiting autosomal recessive disorder in Caucasian population. It due to mutations Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Current symptomatic CF therapies, which treat downstream consequences of CFTR mutations, have increased survival. Better knowledge protein has enabled pharmacologic therapy aiming restore mutated expression and function. These “modulators” revolutionised therapeutic landscape, with potential transform prognosis for a considerable number patients. This review provides brief summary their mechanism action presents thorough results obtained from clinical trials modulators.