Abstract
Cystic fibrosis (CF) is an inherited disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, characterized by both the pulmonary and extrapulmonary symptoms. The drug therapy for pulmonary manifestations of cystic fibrosis targets pnmanly mucostasis, bacterial infection and neutrophilic inflammation.
The treatment of CF extrapulmonary symptoms focuses on the control of pancreatic insufficiency, fat-soluble vitamins deficiency, intestinal obstruction, diabetes, hepatopathy and osteoporosis. CF gene therapy and therapy targeting mutations of the CFTR gene are currently under research.