In cystic fibrosis (CF), the body’s exocrine glands produce abnormal amounts of mucus-like secretions. This has greatest impact in the lungs, pancreas, liver and intestine, resulting in respiratory infections and complications associated with malnutrition.1
Milestones in CF treatment
Since CF was first described in 1938, there have been significant improvements in the median predicted survival age of sufferers: from only 6 months in 1938, through to about 20 years in 1970, and to 37 years in 2009.2,3 CFTR modulators represent a more recent milestone in CF therapy, as drugs in this class actually treat the underlying cause of CF and not just the symptoms.4
Learn more about the synthesis, role and regulation of CFTR channels in epithelial cells: from gene expression to the function of CFTR as an ion channel in the cell membrane.
See an overview of common CFTR mutations and how they influence disease prognosis and clinical management.