Cystic Fibrosis(CF) is a life-threatening disease that affects approximately 30,000 children and adults in the US. CF damages the respiratory and digestive system by producing thick sticky mucus resulting in lung disease and liver disease. Life expectancy is 40 years for a CF patient born today. The CF Foundation is rapidly developing life-extending medications and diligently searching for a cure.
People with CF inherit the defective gene that causes a buildup of thick sticky mucus in the lungs, pancreas, liver, and other organs. When mucus clogs the lungs it can be very difficult to breathe. The thick mucus also traps bacteria in the airways which results in infections and inflammation leading to lung damage and eventually respiratory failure. Respiratory problems are the most serious and persistent complication for people with CF.
The GI system of most CF patients is also compromised by the thick mucus resulting in malabsorption, clogged pancreas, and liver disease. Digestion and weight gain are difficult for most with CF. Diet and food choice requires management on a daily basis with pancreatic enzymes and high fat high calorie diets.
The Cystic Fibrosis Foundation’s fundraising and commitment has enabled the Foundation to support fundamental research in the laboratory that has led to groundbreaking discoveries, including the identification of the gene and protein responsible for cystic fibrosis. By expanding our knowledge of the underlying biology of the disease and its effect on the body, researchers have paved the way for creating new treatments.
The Foundation’s steadfast commitment to advancing CF research has helped enable 12 new treatments for the disease. We have made incredible progress, including the approvals by the U.S. Food and Drug Administration (FDA) of ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), tezacaftor/ivacaftor (Symdeko®), Cayston®, and tobramycin (TOBI®).