Navigation Links
Experimental Drug Offers Hope for Cystic Fibrosis Patients

Ataluren being tested in humans with several diseases caused by genetic mutations

TUESDAY, April 27 (HealthDay News) -- An experimental oral drug called ataluren may treat cystic fibrosis and other diseases caused by genetic "nonsense mutations," U.S. researchers report.

Nonsense mutations disrupt the way cells make proteins. About one-third of gene defects linked to human disease are believed to come from nonsense mutations, and ataluren holds promise for treating more than 2,400 such disorders, the study authors noted.

In cystic fibrosis, the lack of a certain protein results in an imbalance of salt and water in the linings of the lungs and other membranes. In this new study, researchers found that ataluren allowed the protein to be made in mouse cells where it was previously absent, which helped restore normal salt and water balance.

"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 benefit," researcher David Bedwell, a professor in the microbiology department at the University of Alabama at Birmingham, said in a university news release.

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

Bedwell found that ataluren can restore up to 29 percent of normal protein function in mice with cystic fibrosis. The research, funded by PTC Therapeutics Inc., was presented April 26 at the Experimental Biology 2010 conference in Anaheim, Calif.

Ataluren is now being tested in humans with cystic fibrosis, hemophilia A, hemophilia B, Duchenne/Becker muscular dystrophy and other conditions.

More information

The Cystic Fibrosis Foundation has more about cystic fibrosis.

-- Robert Preidt

SOURCE: University of Alabama at Birmingham, news release, April 26, 2010

Copyright©2010 ScoutNews,LLC.
All rights reserved

Related medicine news :

1. Experimental stem cell treatment arrests acute lung injury in mice, study shows
2. Offers Retirees a Smarter Way to Explore Living Options
3. Ovarian cancer study offers vital clues for new therapies
4. American Laser Centers Offers a Solution for Customers Whose Laser Cosmetic Providers Have Gone Out of Business
5. TotalTrans4m Offers Natural Product Just in Time for Wedding Season That Can Jump Start Weight Loss
6. Newly Relaunched Offers Consumers Smart Tips To Consider When Purchasing Car Insurance
7. S.A. Lifeline Foundation Offers New and Improved Website for Those with Pornography Addictions
8. Arsa Offers High Quality Supplements, Cosmetics and Magic Scents from Mexico for Less Than $10
9. Study Offers Insights Into Male Pattern Baldness
10. Extends The March Special SEO Special Offers And Revise Many Services
11. UNC study offers first clinical evidence of anti-cancer drug triggering viral infection
Post Your Comments:
(Date:6/26/2016)... ... June 26, 2016 , ... PawPaws brand pet supplements owned by ... to enhance the health of felines. The formula is all-natural and is made from ... the PawPaws Cat Kidney Support Supplement Soft Chews are Astragalus Root Extract ...
(Date:6/25/2016)... (PRWEB) , ... June 25, 2016 , ... ... health policy issues and applications at AcademyHealth’s Annual Research Meeting June 26-28, 2016, ... work on several important health care topics including advance care planning, healthcare costs ...
(Date:6/25/2016)... ... June 25, 2016 , ... "With 30 hand-drawn hand gesture animations, ... Christina Austin - CEO of Pixel Film Studios. , ProHand Cartoon’s package transforms ... Final Cut Pro X . Simply select a ProHand generator and drag it above ...
(Date:6/25/2016)... ... June 25, 2016 , ... Dr. ... from injury. Recently, he has implemented orthobiologic procedures as a method for treating ... one of the first doctors to perform the treatment. Orthobiologics are substances that ...
(Date:6/24/2016)... ... 2016 , ... A recent article published June 14 on E ... goes on to state that individuals are now more comfortable seeking to undergo not ... as calf and cheek reduction. The Los Angeles area medical group, Beverly Hills Physicians ...
Breaking Medicine News(10 mins):
(Date:6/24/2016)... 2016 Dehaier Medical Systems Ltd. (NASDAQ: ... markets and sells medical devices and wearable sleep respiratory ... strategic cooperation agreement with Hongyuan Supply Chain Management Co., ... June 20, 2016, to develop Dehaier,s new Internet medical ... Dehaier will leverage Hongyuan Supply Chain,s sales platform to ...
(Date:6/24/2016)... June 24, 2016 Research and Markets ... for Companion Diagnostic Tests" report to their offering. ... Companion Diagnostics The World Market for Companion ... medicine diagnostics. Market analysis in the report includes the following: ... (In Vitro Diagnostic Kits) by Region (N. America, EU, ROW), ...
(Date:6/24/2016)... 2016   Pulmatrix, Inc ., (NASDAQ: ... drugs, announced today that it was added to the ... its comprehensive set of U.S. and global equity indexes ... important milestone for Pulmatrix," said Chief Executive Officer ... our progress in developing drugs for crucial unmet medical ...
Breaking Medicine Technology: