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Health Beat: Cystic fibrosis breakthrough

By Melanie Falcon, Anchor / Reporter, @Melanie_Falcon, MFalcon@wfmz.com
Published On: Mar 07 2013 12:01:08 PM CST
Updated On: Mar 11 2013 04:35:46 PM CDT

Each year, more than 1,000 children are born with cystic fibrosis. Kids with it are not expected to live past their 30s, but now that could be changing.

Each year, more than a thousand children are born with cystic fibrosis. Kids with it are not expected to live past their 30s, but now that could be changing.

The FDA has recently approved a new drug to help kids fight cystic fibrosis. Kalydeco is for children over 6, and doctors said it's not only taking away the symptoms, but increasing their life expectancy.

Rylee is a girl on the go. She won't let her disease slow her down.

"I have cystic fibrosis," Rylee said.

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.

"Any kind of plan that we had for the future or dream that we had for our daughter was completely shattered," said Amy, Rylee's mom.

A year ago, Rylee was taking 20 pills a day. Now, she's taking only 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.

"And that infection can fester because it just doesn't move," said Dr. Carolyn Cannon, director of the Pediatric Cystic Fibrosis Care and Teaching Center, Children's Medical Center at Dallas.

Kalydeco restores the function of the protein, clearing out the lungs, and relieving symptoms of the disease.

"It is absolutely a game changer. It changes their life," said Cannon.

Giving kids like Rylee a chance for a long, healthy life.

"I can dream anything for her now," said Amy.

The drug is expensive, but some insurance carriers, like Rylee's, cover much of it.

DOWNLOAD and VIEW research summary

DOWNLOAD and VIEW the full-length interview with Dr. Carolyn Cannon about the new breakthrough for cystic fibrosis.