Cystic Fibrosis: an Update on Disease Pathophysiology, Management, and Novel Modalities of Therapy

Abstract

To explain the underlying pathophysiology of cystic fibrosis and to describe current treatment modalities for disease manifestations, including an introduction to novel therapies known as CFTR modulator drugs, which are now available to over 50% of people with cystic fibrosis. CFTR modulator drugs, aimed at restoring CFTR protein function, have been shown to improve lung function and reduce pulmonary exacerbations, improve BMI and QOL, and are a new added treatment to the traditional therapies for cystic fibrosis. With the current modalities of treatment available to patient with cystic fibrosis, the quality of life and the life expectancy continues to improve. Future directions for research include looking at novel anti-inflammatory agents, antimicrobial agents, mucociliary clearance agents, and newer generation CFTR modulator drugs/drugs that restore CFTR function.