Navigation Links
PTC124 Shows Promising Activity in Cystic Fibrosis; Phase 2 Proof-of-Concept Data Published in the Lancet
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

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
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/1/2014)... October 01, 2014 The U.S. Department ... Southern Illinois University Edwardsville as part of the Trade ... The funding is co-administered by the Department of Labor ... Secretary of Labor Thomas E. Perez and Secretary of ... in job-driven training grants to nearly 270 colleges across ...
(Date:10/1/2014)... -- Industry leaders from retail pharmacies ... the theme, ,Commercial Opportunities in the Era of Reform, ... Elsevier , a world-leading provider of scientific, technical ... gathering of some of the industry,s most prominent healthcare ... and in the future. The Elsevier Healthcare ...
(Date:9/30/2014)... September 30, 2014 Applying a well-known optical ... at Harvard University has demonstrated the ability to ... work that holds promise for making future, flexible ... the sides of next-gen rocket ships and spacecraft ... passes through oil in water, it becomes iridescent, ...
(Date:9/30/2014)... a bio-mimicking analog of one of nature,s most ... research team led by Alejandro Briseno of the ... step in developing long-sought polymer architecture to boost ... in electronic devices. , Briseno, with colleagues ... at Stanford University and Dresden University of Technology, ...
Breaking Biology Technology:U.S. Dept. of Labor Awards SIUE Nearly $10-Million Grant 2U.S. Dept. of Labor Awards SIUE Nearly $10-Million Grant 3U.S. Dept. of Labor Awards SIUE Nearly $10-Million Grant 4Elsevier Healthcare and Pharmaceutical Economics Conference 2014 To Convene Industry Leaders to Discuss Top Healthcare Business Issues 2Elsevier Healthcare and Pharmaceutical Economics Conference 2014 To Convene Industry Leaders to Discuss Top Healthcare Business Issues 3Taking thin films to the extreme 2Blades of grass inspire advance in organic solar cells 2Blades of grass inspire advance in organic solar cells 3
... , SOUTH SAN FRANCISCO, Calif., Sept. ... announced the pricing of an underwritten public offering of 13,000,000 shares ... $7.25 per share. All of the shares are being offered by ... additional 1,950,000 shares to cover over-allotments, if any. The offering is ...
... , , ... ITMN ) today announced that it will be collaborating ... the research of inherited genetic factors that may play a role ... National Jewish with access to DNA collected from patients who participated ...
... , EXTON, Pa., Sept. 16 Kensey Nash ... the 2009 UBS Global Life Sciences conference at the Grand Hyatt ... , , Joseph W. Kaufmann, Kensey Nash,s President ... p.m. (ET). To hear the live webcast of the Kensey ...
Cached Biology Technology:Rigel Announces Pricing of Public Offering of 13,000,000 Shares of Common Stock 2InterMune Announces Collaboration With National Jewish Health on NIH-Supported Genetic Research in Idiopathic Pulmonary Fibrosis 2InterMune Announces Collaboration With National Jewish Health on NIH-Supported Genetic Research in Idiopathic Pulmonary Fibrosis 3
(Date:9/30/2014)... visit the Mediterranean each year, but its deep-blue ... Earth. Almost 1,000 alien species, including fish, crustaceans, ... through human activities. In the open-access journal ... of researchers analyzed data from a new information ... how the introduction of alien species has changed ...
(Date:9/30/2014)... and strawberries all have in common? Unlike most ... and are diploid, these organisms are all polyploid, meaning ... and salmon have 3 and 4 sets of chromosomes, ... seem surprising, in fact most plant species are polyploid. ... century ago, but only recently, with the development of ...
(Date:9/30/2014)... drinking water may increase the risk of stillbirth ... led by a Boston University School of Public ... journal Environmental Health , compared 1,091 PCE-exposed ... in Cape Cod, Ma., where water was contaminated ... by the installation of vinyl-lined asbestos cement pipes. ...
Breaking Biology News(10 mins):Biodiversity in the Mediterranean is threatened by alien species 2Gene doubling shapes the world: Instant speciation, biodiversity, and the root of our existence 2Gene doubling shapes the world: Instant speciation, biodiversity, and the root of our existence 3Gene doubling shapes the world: Instant speciation, biodiversity, and the root of our existence 4Contaminated water linked to pregnancy complications, BU study finds 2
... European researchers have published a study which analyses the health ... of osteoporotic fractures in the population aged over 50. ... that links nutrition and fracture risk, and health economics. It ... countries which have varying levels of dairy product intake in ...
... dynamic role cilia play in guiding the migration of ... structures on the surfaces of cells, but here they ... mouse embryos, researchers were able to see cilia extending ... be receiving signals needed for neurons to find their ...
... for a boat ride or even taken a drink from the ... how valuable? That,s always been a tough question for policy makers ... societal needs that compromise it, such as the need to grow ... so has been greatly enhanced by a new policy-making framework developed ...
Cached Biology News:New health-economic model shows benefits of boosting dietary calcium intake 2Cilia guide neuronal migration in developing brain 2Study offers new tool for incorporating water impacts into policy decisions 2
Lysozyme C (W-20)...
Request Info...
Abgene's ALTO-8 is the first automated decapper/capper for Abgene Twist-Lock tubes....
... X-Gal (5-bromo-4-chloro-3-indoyl-beta-D-galactopyranoside) is a ... identify recombinant plasmids. In ... and IPTG, lac ... blue, whereas recombinant colonies ...
Biology Products: