Drug approved to treat cystic fibrosis' root cause

[xchrom]

http://hosted.ap.org/dynamic/stories/U/US_CYSTIC_FIBROSIS_NEW_DRUG?SITE=AP&SECTION=HOME&TEMPLATE=DEFAULT&CTIME=2012-01-31-11-57-23

WASHINGTON (AP) -- The first drug that treats the root cause of cystic fibrosis won approval Tuesday, offering a life-changing treatment for a handful of patients with the deadly illness and broader hope for thousands more patients with the inherited disease.

About 30,000 Americans live with cystic fibrosis, a disease that causes sticky mucus buildup in the lungs and other organs, leading to infections, digestive problems and death in young adulthood. The typical life expectancy is about 37 years, according to the Cystic Fibrosis Foundation.

The Food and Drug approved Vertex Pharmaceuticals Inc.'s Kalydeco for patients with a rare form of the disease that affects just 1,200 people in the U.S., about 4 percent of affected population nationwide. These patients have a protein defect that prevents their cells from properly absorbing and excreting salt and water. Studies of the drug showed it significantly improved lung function and reduced other symptoms of cystic fibrosis.

"Even though this drug isn't for the majority of people, it proves that you can look at the mistake in the genes and design a drug in a rational way that will fix the problem," said Dr. Drucy Borowitz of the State University of New York at Buffalo, where she directs the cystic fibrosis program.

[hedgehog]

1. Just like to point out that UB, my alma mater,

is a publicly funded, state school.

http://www.buffalo.edu/

[xchrom]

2. that's true! -- i hadn't thought about that. very cool. nt

[Lawlbringer]

3. Cue Alex Jones saying it's toxic mind control

in 5...4...3...2...

[HuckleB]

4. K&R.