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:7/24/2014)... rely on sophisticated equipment, trained personnel, and detection ... against terrorist attacks. A revolutionary new electronic chip ... their job much easier. , The groundbreaking nanotechnology-inspired ... Aviv University ,s School of Chemistry and Center ... Herzliya company Tracense, picks up the scent of ...
(Date:7/24/2014)... synthetic processes prefer to settle into equilibriuma state ... within the realm of non-equilibrium conditions where new ... energy and phases, such as temperature fluctuations, freezing ... to regulate their body temperature, airplanes to fly, ... , But even though these conditions exist ...
(Date:7/24/2014)... 2014 SRI International has been awarded a ... of Allergy and Infectious Diseases (NIAID), part of the ... potential therapies for HIV infection and AIDS. The contract ... AIDS and the complications and opportunistic infections associated with ... transmission of HIV. According to the ...
(Date:7/24/2014)... , July 24, 2014  Neogen Corporation (Nasdaq: ... future forecasts of its potential revenue from new rodenticide ... to investors and analysts on July 22, 2014, Neogen,s ... analysts regarding this research. "It was my ... about the potential of a new type of rodenticide, ...
Breaking Biology Technology:Nano-sized chip 'sniffs out' explosives far better than trained dogs 2New approach to form non-equilibrium structures 2National Institute of Allergy and Infectious Diseases Awards SRI International Contract to Study New Therapies for HIV and AIDS 2National Institute of Allergy and Infectious Diseases Awards SRI International Contract to Study New Therapies for HIV and AIDS 3Neogen comments on SenesTech 2
... Reaches severance agreement with unionsSEATTLE, May 15 Cell ... that, as previously discussed in a press release on ... operations and has reached agreement with the employee union ... a reduction of CTI,s total headcount in Italy by ...
... (TSX:ONC, NASDAQ: ONCY ) announced today that data from ... at the 2009 American Society of Clinical Oncology (ASCO) Annual ... 29-June 2, 2009. The abstracts are available on the ASCO ... poster presentations, - Dr. Monica ...
... May 14 /PRNewswire-FirstCall/ - Spectral Diagnostics Inc., (TSX: SDI), ... and a therapeutic targeting severe sepsis, today announced its ... 2009. , "This quarter, we achieved an important milestone ... Toraymyxin(TM) for the US market after thorough due diligence ...
Cached Biology Technology:Cell Therapeutics Closes Italian Facility, Reduces Headcount and Expects to Cut $14 Million in Operating Expenses 2Oncolytics Biotech(R) Inc.'s Research Collaborators to Present Data on REOLYSIN(R) Clinical Trials at ASCO Conference 2Spectral announces first quarter 2009 results 2Spectral announces first quarter 2009 results 3Spectral announces first quarter 2009 results 4Spectral announces first quarter 2009 results 5Spectral announces first quarter 2009 results 6Spectral announces first quarter 2009 results 7
(Date:7/24/2014)... York, NY - July 24, 2014 -- Keryx Biopharmaceuticals, ... from the long-term, randomized, active control Phase 3 study ... iron-based phosphate binder, for the treatment of hyperphosphatemia in ... PERFECTED study (PhosphatE binding and iRon delivery with FErric ... Journal of the American Society of Nephrology ...
(Date:7/24/2014)... Bushmeat, the use of native animal species for food ... be a significant factor in the decline of many ... new study indicates that more than half of the ... raptors and hornbills., "By surveying not only the meat ... being eaten inside the forest by hunters and brought ...
(Date:7/24/2014)... study has identified two factors that characterize sustainable ... production of highly qualified physics teachers. Specifically, one ... physics teacher education in combination with institutional motivation ... viable. Science, Technology, Engineering and Math (STEM) teacher ... study points the way for institutions seeking to ...
Breaking Biology News(10 mins):Zerenex™ (ferric citrate) long-term Phase 3 study results published in JASN 2Zerenex™ (ferric citrate) long-term Phase 3 study results published in JASN 3Zerenex™ (ferric citrate) long-term Phase 3 study results published in JASN 4Creating sustainable STEM teacher preparation programs 2
... noses and Microsoft Windows, infamous blue screen of death ... being made by a new research report appearing online ... Specifically, scientists now know exactly why we sneeze, what ... not work properly. Much like a temperamental computer, our ...
... Olympic athletes may be the best trained and physically ... susceptible to back pain. BackPainRelief.net is releasing an infographic ... injuries can be avoided. (Photo: ... for a number of injuries, and back pain is ...
... (July 30, 2012) When the longest cells in the body ... is no easy task. This is in part because these ... one meter in adult humans which is a lot ... order to reach the "command center" of the cell,s nucleus. ...
Cached Biology News:Allergies? Your sneeze is a biological response to the nose's 'blue screen of death' 2Backpainrelief.net Tackles Sports Injuries with Infographic 2Long-distance distress signal from periphery of injured nerve cells begins with locally made protein 2Long-distance distress signal from periphery of injured nerve cells begins with locally made protein 3
... IndicationIncludes all the features of Tracker, plus software ... for a user defined period of time in ... a family of instruments all built around a ... and that can track two fields at once ...
... all features of Tracker, plus software to note ... and provide a food reward.The Spatial Behavior line ... a video camera, frame grabber, and feature extraction ... once and up to 6 moving animals. ...
... Place AvoidanceIncludes all features of Tracker, plus ... a user defined subsection of the arena, and ... family of instruments all built around a video ... that can track two fields at once and ...
tracking dye in electrophoresis; free acid, C 19 H 10 BrO 5 S; MW: 669.96...
Biology Products: