Abstract

The field of cystic fibrosis (CF) continues to evolve at a fast pace thanks to novel observations that have enabled deeper understanding of the disease pathophysiology. Parallel groundbreaking developments in innovative therapies permit, for the first time, distinct disease modification.This review highlights important discoveries in fluid homeostasis and mucus secretion in CF that further informs the pathophysiology of the airway disease that characterizes CF. In addition, current concepts and novel paradigms, such as 'theratypes' and 'CF transmembrane conductance regulator chaperome', which will be important for the continued development of disease modifying therapies, are reviewed.The rate of progress in the field continues to accelerate with new knowledge informing the development of innovative therapies. This has already led to tangible substantial and unprecedented clinical benefit for selected subsets of the CF patient population. In the years ahead, further knowledge acquisition may motivate the extension of these benefits to the larger population of people with CF.

View details for DOI 10.1097/MOP.0000000000000226

View details for Web of Science ID 000354214800009

View details for PubMedID 25888148