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New experimental drug offers hope

According to a recent report published by the University of Alabama at Birmingham, researchers are closer to finding a drug that will treat cystic fibrosis.

Cystic fibrosis is caused by genetic "nonsense mutations" that disrupts the way the human cells make protein.The new drug, called ataluren, is said to fix the disruption by allowing the protein to be made in mouse cells where it was previously not present.

According to researchers at UAB, the drug will also be used to treat over 2,400 other genetic diseases that are caused by "nonsense mutations."

"When you treat a genetic disease, the bottom line is how much of the missing protein do you need to restore to have a therapeutic benefits," researcher David Bedwell, a professor in the microbiology department at UAB, said in a university news release.

"It comes down to the threshold of protein rescue. For some diseases, it might be 1 % of the protein you need restored, and for other disease you may need 50 % of protein restored," he explained.

Bedwell found that ataluren restored up to 29 % of normal protein function in mice with cystic fibrosis. The drug is currently being tested on humans.

March 10, 2011 at 3:18 pm
(1) Alyssa says:


July 18, 2011 at 12:31 pm
(2) Rob says:

This is great news I would like to check back to see the progress

September 26, 2011 at 7:54 pm
(3) Le Troll says:


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