Cystic Fibrosis

Cystic Fibrosis ("CF") is a chronic, life-threatening, genetic disease caused by inheriting two dysfunctional copies of the CFTR gene. CF affects approximately 30,000 patients in the U.S. and 75,000 patients worldwide. People with CF have thick, sticky mucus that clogs their airways with chronic inflammation and recurrent bacterial infections in their lungs. In the gastrointestinal tract, they also have mucus accumulation, inflammation, and bacterial overgrowth. Dysfunctional CFTR genes cause an exaggerated but also ineffective inflammatory response that damages tissue and can be compounded by infection. CF results in destruction of lung tissue, lung fibrosis, pancreatic insufficiency, CF-related diabetes, malabsorption, malnutrition, growth retardation, and liver disease, including cirrhosis. The harmful inflammation and accompanying fibrosis in CF damages multiple organs, impairs organ function, reduces health-related quality of life, and can lead to death. For more information, please refer to the Cystic Fibrosis Foundation:

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