New drugs approved to treat Cystic Fibrosis

Posted at 8:56 PM, May 18, 2015
and last updated 2015-05-19 09:52:36-04

Two newly approved drugs show great promise in treating Cystic Fibrosis. The medications have been shown to be very effective in many patients, creating a chance for parents to see their children enjoy their lives.

Cystic Fibrosis prevents fluid from moving through the lungs, causing severe and often fatal congestion. The usual prognosis for affected children is not good. Many lose their battle with CF before attending grade school.

But the two new drugs have spawned new hope for patients and their families.

“These are the first drugs of their kind that treat the disease itself and not just the symptoms. So that’s what's so exciting about it,” said Laura Hadley, executive director of the Cystic Fibrosis Foundation.

Medications Kalydeco and Orkami are not a cure but rather maintenance drugs that can significantly reduce the severity of CF and prolong life expectancy. Kalydeco in particular has had a profound effect on many young patients.

“It’s a game changer,” Hadley said. “People’s lives have been significantly impacted that have the correct mutation to be able to benefit from this drug.”

Two-year-old Penny Hepworth of Kaysville is one such patient. Penny’s mom spoke to FOX 13 News about the fears any parent faces with the news their child has been diagnosed with CF.

Watch the full story in the video above.

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