Cystic Fibrosis (CF) is a genetic disease that results from mutations in a single gene (called CFTR, cystic fibrosis transmembrane conductance regulator). It is a recessive condition, which means that patients living with the condition carry two copies of the faulty gene. CF causes problems in multiple organs. For most patients, the greatest health difficulties are caused by progressive lung disease, resulting from chronic bacterial infection.
Researchers have developed therapies to correct the faulty, mutated protein in people with CF. These are called CFTR modulators. These have been shown to improve patients' lung function and overall quality of life. However, each type CFTR modulator is designed for a specific mutation, and so some patients are unable to receive them due to their specific mutation. Researchers are working to develop a gene therapy that could be used to treat all mutations causing CF.
In this section you will find factsheets about CF and about ongoing research in this area, as well as guidelines as to where you can look for support if you or a loved one are living with CF.
