Navigation Links
Experimental Drug Offers Hope for Cystic Fibrosis Patients
Date:4/27/2010

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. RetiringbyDesign.com 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 Autoinsurance.net 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. SEO-Peace.com 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:
*Name:
*Comment:
*Email:
(Date:4/29/2016)... ... April 29, 2016 , ... Shamangelic Healing, ... addition Onnit brand Alpha BRAIN and New Mood Daily-Stress Formula for brain optimization ... optimization products to the store is just one more way Shamangelic Healing supports ...
(Date:4/29/2016)... ... April 29, 2016 , ... The White House announced efforts ... more information about their loan terms and accounts, and more protections for borrowers. ... federal and private loans, has reached $1.3 trillion, with 43 million Americans holding ...
(Date:4/29/2016)... ... April 29, 2016 , ... ... team BioCellection won the $30,000 Perlman Grand Prize of the 2016 Wharton Business ... Award, the Michelson People’s Choice Award, and the Committee Award for Most ‘Wow ...
(Date:4/29/2016)... ... April 29, 2016 , ... Dr. Robert Mondavi, one of the dentists ... dentistry is a fast-growing field as more patients are discovering the many different ways ... about the options currently available to them and which ones might work for their ...
(Date:4/29/2016)... Jupiter, FL (PRWEB) , ... April 29, 2016 ... ... it will feature Grassland Dairy Products, Inc. in an upcoming episode, airing third ... than a century of churning cream into butter, Grassland Dairy Products, located in ...
Breaking Medicine News(10 mins):
(Date:4/27/2016)... , le 27 Avril 2016 ... a progressé de +5% sur le trimestre, soutenu ... de consommables  Croissance de +16% des ... Mauna Kea Technologies (Euronext : MKEA, FR0010609263 ; ... confocale laser, annonce aujourd,hui son chiffre d,affaires pour ...
(Date:4/27/2016)... York , April 27, 2016 ... report titled, "Skincare Devices Market - Global Industry Analysis, ... According to the report, the global skincare devices market ... is anticipated to expand at a CAGR of 10.1% ... in 2023. Browse the full Skincare Devices Market ...
(Date:4/27/2016)... Kan. , April 27, 2016  Bayer ... , a senior from the University of Florida ... of the Bayer Excellence in Communication Award (BECA). ... schools, which were awarded a total of $70,000 ... the last four years, Bayer has provided a ...
Breaking Medicine Technology: