What is Cystic Fibrosis? No cure yet for inherited disease

Cystic Fibrosis is an inherited disease that causes thick, sticky mucus to form in the lungs, pancreas and other organs. In the lungs, this mucus blocks the airways, causing lung damage and making it hard to breathe. In the pancreas, it clogs the pathways leading to the digestive system, which interferes with proper digestion and results in vitamin deficiency and malnutrition.

For a child to get cystic fibrosis, both parents must carry the gene that causes it. In the United States, CF is most common among Caucasians; one out of every 20 people is a carrier, and 1 of every 2,000 to 3,000 babies is born with CF. People with CF have a shorter-than-normal life expectancy, but the good news is that as treatments for CF improve, life expectancy is rising and many now live into their 30s, 40s and beyond.

While there is no cure yet, a breakthrough in treatment was approved by the FDA in January. Kalydeco, a new oral medication, was developed with significant support from the CF Foundation. Patients taking Kalydeco had improved lung function (and a reduction in coughing and shortness of breath), fewer infections and they gained weight.

Sources: Cystic Fibrosis Foundation, American Lung Association


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