PTC124 Shows Promising Activity in Cystic Fibrosis; Phase 2 Proof-of-Concept Data Published in ...(vercoming nonsense mutations as thebasis...)

Date:8/20/2008

vercoming nonsense mutations as the basis for treating genetic disease," said Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics. "Based on these results, we intend to initiate a Phase 2b study later this year to evaluate the clinical benefit of PTC124 in adults and children with nonsense-mutation-mediated CF. Given the potential applicability of PTC124 to multiple genetic disorders, we have a pivotal study of PTC124 for nonsense-mutation Duchenne/Becker muscular dystrophy ongoing and are planning proof-of-concept studies in additional genetic disorders."

The paper entitled "Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial" is available in an advanced online publication of Lancet on Thursday, August 21st (http://www.lancet.com).

About Cystic Fibrosis

Cystic fibrosis (CF) is a life-threatening genetic disease that causes serious lung infections and digestive complications. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and nearly 70,000 people worldwide. There is a commercially available genetic test to determine if a patient's CF is caused by a nonsense mutation, and it is estimated that nonsense mutations are the cause of CF in approximately 10 percent of patients in the United States and Europe and over 50 percent of patients in Israel. There is currently no available therapy to correct defective CFTR production and function. Instead, available treatments for CF are designed to alleviate the symptoms of the disease. These treatments include chest physical therapy to clear the thick mucus from the lungs, antibiotics to treat lung infections, and a mucus-thinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to a
'/>"/>

SOURCE PTC Therapeutics, Inc. Copyright©2008 PR Newswire. All rights reserved

1 GOOD

Page: 1 2 3 4 5
Related biology technology :

1. PTC124 Featured at Third Annual Congress of Myology
2. PTC Therapeutics Announces Encouraging Additional Phase 2 Results of PTC124 in Cystic Fibrosis
3. Asthma Uncontrolled in More Than 40 Percent of Adults With the Disease, New Landmark Survey Shows
4. Atrium shows a strong financial performance for its first quarter
5. A Smoking Gene: deCODE Shows How a Single Variant in the Sequence of the Genome Confers Nicotine Dependence and Risk of Lung Cancer and Other Disease
6. Cardiums Corgentin Preclinical Program Shows High Levels of Targeting to Acutely Ischemic Heart Muscle Following Intracoronary Infusion
7. Survey Shows Most Women Cant Separate Fact From Fiction When It Comes to Cervical Cancer Prevention
8. UW paper in Science shows how some solids mimic liquids on nanoscale
9. New research shows that iPods do not interfere with cardiac pacemakers
10. New kind of transistor radios shows capability of nanotube technology
11. Study Shows Coronado Biosciences Bcl-2 Inhibitor, Apogossypol, is More Efficacious, Less Toxic than Gossypol in Animal Models