How does Kalydeco work to treat cystic fibrosis?

Kalydeco (ivacaftor) works to treat a form of cystic fibrosis (CF) caused by a certain mutation (6551D) in a specific gene. The gene is called the transmembrane conductance regulator. Kalydeco works by improving the function of a protein in the body in people who have this gene mutation. By improving the function of this protein, Kalydeco prevents some of the build-up of thick mucus in the lungs that is a hallmark of CF and helps with other symptoms as well.

Kalydeco is the first medication that treats the underlying cause of cystic fibrosis. However, it does not treat the most common form of CF.

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