Kalydeco, a brand new drug that could extend the lives of those fighting a rare form of cystic fibrosis, has been approved by the FDA. Made by Vertex Pharmaceuticals, there are about 70,000 people worldwide who have the kind of cystic fibrosis it can treat.
FOX News Radio's Lisa Brady has more:
(Borowitz) "This drug is so remarkable."
...The first to zero-in on the mucus buildup in the lungs that leads to infections in the lungs and, ultimately, early death. Dr. Drucy Borowitz, who heads the cystic fibrosis program at the University of Buffalo, says after taking two pills a day for two weeks, patients she studied showed significant improvement in lung function.
(Borowitz) "Their sweat test had gone so low that we would not have diagnosed them with cystic fibrosis. That's how dramatic the drug is."
But it isn't for all cystic fibrosis patients. In the US, only about 1200 have the specific gene mutation it targets.
(Borowitz) "It is the promise of the Human Genome Project that you could look at a specific mutation and fix it."
And that could bode well for battling other diseases.
Lisa Brady, FOX News Radio.