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Cystic Fibrosis Breakthrough
Each year, a thousand children are born with cystic fibrosis. Kids with it are not expected to live past their thirties, but now many could live much longer.
Rylee is a girl on the go. She won't let her disease slow her down. "I have cystic fibrosis," she admits. Cystic fibrosis causes the body to produce a thick mucus that clogs the lungs and pancreas, making it difficult to clear the lungs and break down food. A child with CF constantly fights infections and to maintain a healthy weight.
Rylee's mom Amy said, "Any kind of plan that we had for the future or dream that we had for our daughter was completely shattered."
A year ago, Rylee was taking 20 pills a day. Now, she's only taking one!
Kalydeco is the first drug that targets the underlying cause of CF, the defective protein called CFTR. In some patients, this protein does not allow fluids to clean the surface of the lungs. That can cause deadly infections.
Dr. Carolyn Cannon of the Cystic Fibrosis Care Center said, "And that infection can fester because it just doesn't move." Kalydeco restores the function of the CFTR protein, clearing out the lungs, and relieving symptoms of the disease.
"It is absolutely a game changer. It changes their life."
Giving kids like rylee a chance for a long, healthy life.
"I can dream anything for her now."
Not only does kalydeco take away the symptoms for some kids with cystic fibrosis, doctors believe it might allow them to live decades longer than expected. Kalydeco was recently FDA approved for kids over six.
Rylee is using it off-label and is the first child under the age of six to start on it. The drug costs $300,000 a year, but Rylee's insurance pays all but 90 dollars a month.