Navigation Links
PTC Therapeutics Announces Publication of Preclinical Data in PNAS
Date:2/4/2008

ng the premature stop signal and reading through the complete genetic instructions, resulting in the restoration of a full-length, functional protein. Patients with CF lack the CFTR protein, a chloride channel that maintains proper hydration of epithelial cells in the lung, pancreas, and liver. The data in PNAS demonstrate that PTC124 allows CFTR to be made in cells in which it was previously absent, to be delivered to the proper cellular location, and to induce chloride channel function. Collectively, these results indicate that this new approach may ultimately be applicable to a subset of patients across a large number of genetic disorders.

The paper entitled, "PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model," will be available in an advanced online publication of PNAS during the week of February 4, 2008 (http://www.pnas.org/papbyrecent.shtml). This publication is the result of collaborative efforts between the University of Alabama at Birmingham and PTC Therapeutics.

"These preclinical data further demonstrate the potential applicability of the PTC124 mechanism of action to multiple genetic disorders," said Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics. "We look forward to further evaluating PTC124 in registration-directed clinical trials in both CF and DMD this year, as well as in studies in additional genetic disorders in the future."

About PTC124

PTC124 is an orally delivered investigational new drug in Phase 2 clinical development for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process, producing a shortened, non- functional protein. PTC124 has restored production of full-length, functional proteins in preclinical genetic disease m
'/>"/>

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

Page: 1 2 3 4

Related medicine technology :

1. ImaRx Therapeutics Transitions Business Strategy
2. Protalix BioTherapeutics Extends Research Agreement With Yissum Based on Promising Acetylcholinesterase Program Results in Animal Study
3. Fundamental Applied Biology, Inc. Is Awarded SBIR Phase IIB Grant for the Commercialization of Protein Therapeutics Using Cell Free Technology
4. Juvaris BioTherapeutics Announces Cooperative Research and Development Agreement With The Centers for Disease Control and Prevention
5. Halozyme Therapeutics Announces Peer-Reviewed Publications of the INFUSE-LR Clinical Trial Results and Clinical Practice Experience With Hylenex
6. William R. Ringo Joins Hyperion Therapeutics, Inc. Board of Directors as Chairman
7. Tacere Therapeutics Enters Collaboration and License Agreement With Pfizer To Develop and Commercialize RNAi Hepatitis C Drug
8. Celldex Therapeutics Receives Fast-Track Designation for CDX-110, a Novel EGFRvIII Vaccine for Glioblastoma
9. Amicus Therapeutics to Present at the 26th Annual JPMorgan Healthcare Conference
10. Interim Safety Analysis Supports Continuation of Allos Therapeutics Pivotal Phase 2 PROPEL Trial of PDX in Patients with Peripheral T-cell Lymphoma
11. Northwest Biotherapeutics Conducting Ovarian Cancer Clinical Trial With DCVax(R)-L at University of Pennsylvania
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/28/2015)... , August 28, 2015 The report "Eubiotics ... (Pork/Swine, Poultry, Ruminant, and Aquaculture), & by Region ( ... Asia-Pacific , and Rest of the World) - ... global market for Eubiotics was valued at USD 4.62 Billion ... by 2020, at a CAGR of 7.4% from 2015 to ...
(Date:8/28/2015)... and LONDON , August 28, 2015 /PRNewswire/ ... approach seamlessly connects people, technologies and ... improving outcomes and the cost of healthcare ... AEX: PHIA) today announced its presence at  ESC Congress 2015 ... including Heart Model A.I. , EchoNavigator and IntelliSpace Cardiovascular, that ...
(Date:8/27/2015)... ALEXANDRIA, Va. , Aug. 27, 2015 /PRNewswire-USNewswire/ ... in the Food and Drug Administration,s (FDA,s) draft ... including reference products and biosimilars, to bear a ... years, AMCP has been seeking a decision from ... joined 18 diverse health care stakeholders urging the ...
Breaking Medicine Technology:Eubiotics Market Worth 7.05 Billion USD by 2020 2Eubiotics Market Worth 7.05 Billion USD by 2020 3Eubiotics Market Worth 7.05 Billion USD by 2020 4Eubiotics Market Worth 7.05 Billion USD by 2020 5Philips Showcases New Integrated Cardiology Solutions to Advance Minimally Invasive Procedures at ESC Congress 2015 2Philips Showcases New Integrated Cardiology Solutions to Advance Minimally Invasive Procedures at ESC Congress 2015 3Philips Showcases New Integrated Cardiology Solutions to Advance Minimally Invasive Procedures at ESC Congress 2015 4AMCP Is Disappointed in FDA's Draft Guidance and Proposed Rule Calling for Suffix on Nonproprietary Names of Biological Products 2
... ADDISON, Texas, May 11, 2011 ULURU Inc. ... scheduled a conference call to discuss the first quarter ended March ... on Tuesday, May 17, 2011 at 9:00 a.m. Eastern Time. ... for international callers dial (850) 429-1388 (access code 23748#) five to ...
... May 11, 2011 HCB Health, one of the ... and the leading healthcare agency in the Southwest, won ... the 22nd annual Manny Awards, a national competition honoring ... for a consumer campaign supporting Sound Surgical Technologies, VASER® ...
Cached Medicine Technology:ULURU Inc. Announces Conference Call to Discuss Financial Results for the First Quarter Ended March 31, 2011 and to Provide a Business Update 2HCB Health Wins Manny Award for Best Medical Device Campaign 2
(Date:8/29/2015)... ... 29, 2015 , ... People who have heart or lung ... asthma, are at higher risk from wildfire smoke especially in Calgary, Alberta.Those with ... problems (arrhythmia or irregular heartbeat) are also sensitive to air pollution. , ...
(Date:8/29/2015)... ... 29, 2015 , ... The popular television program VOICES IN AMERICA, hosted by ... will be devoting an entire short segment to the subject of diabetes. Diabetes is ... on a daily basis, but recent advancements in medical science may prove to be ...
(Date:8/28/2015)... (PRWEB) , ... August 29, 2015 , ... Rio ... higher learning and the student experience as part of the EDUCAUSE - Next Generation ... Foundation . , This is the third Breakthrough Models Incubator hosted by ...
(Date:8/28/2015)... ... August 28, 2015 , ... An online provider of CPAP machines, ... purchase of CPAP equipment more affordable by offering shipping at no cost to you, ... products and orders of any size. My Cpap Store is located in Las Vegas, ...
(Date:8/28/2015)... ... 2015 , ... The Kentucky State Department of Workforce Investment Commissioner Beth Kuhn ... applying for, they are unable to pass the initial drug test and therefore unable ... in order to be able to hire long-term, skilled talent. (1) Will Wesch, Director ...
Breaking Medicine News(10 mins):Health News:Heart Fit Clinic Sounds the Alarm on Forest Fire Smoke and Chronic Heart Disease in Calgary Area 2Health News:Rio Salado College Joins 9 Educational Leaders to Reinvent Higher Learning 2Health News:Rio Salado College Joins 9 Educational Leaders to Reinvent Higher Learning 3Health News:Rio Salado College Joins 9 Educational Leaders to Reinvent Higher Learning 4Health News:My Cpap Store Now Offering Delivery Special 0 Cost to You 2Health News:Hiring Obstacles: Drug Abuse a Problem for Workforce 2Health News:Hiring Obstacles: Drug Abuse a Problem for Workforce 3Health News:Hiring Obstacles: Drug Abuse a Problem for Workforce 4
... NEW YORK, Feb. 11 New research suggests proper ... your heart health. About 8 in 10,adults have experienced ... of gum disease and the first stage of gum ... Listen to this report from Colgate at:, ...
... health,professionals are joining forces at the 2nd Annual ... April 3-6, 2008 in,Taos, New Mexico. Research and ... most pertinent health issues including celiac disease, gluten,sensitivity, ... Based on extensive medical research, IHI has determined ...
... who could benefit most have the poorest eating habits, ... with high blood pressure follow recommended dietary guidelines that ... In fact, people with hypertension actually appear to be ... according to the report published in the Feb. 11 ...
... State-by-state survey also found prevalence rate among men not as ... -- Uncontrolled hypertension rates are on the increase among American ... heart disease and stroke among American men is still not ... , "Blood pressure that is higher than optimal is among ...
... American Academy of ... Dermatology, ... that validates the benefits of alpha-hydroxy acid (AHA),at- home peels in ... as lasers and microdermabrasion. The,data were highlighted in the Poster Session ...
... start ... families, CHARLOTTE, N.C., ... Endocrinology Associates of,Charlotte (REACH) eyeing far more aggressive means to recruit young ... --,in efforts to stem an alarmingly widening gap between supply and demand., ...
Cached Medicine News:Health News:Conference to Explore America's Health Challenges 2Health News:Most With High Blood Pressure Dont Follow Recommended Diet 2Health News:High Blood Pressure on Increase Among American Women 2Health News:NeoStrata Research Validates Anti-Aging Benefits of At-Home AHA Peels As Complement to In-Office Non-Invasive Cosmetic Procedures 2Health News:Charlotte IVF Experts Issue Urgent Call for Egg Donors, Eye New Recruiting Tools 2
... Impulse II lead series are silicon ... extend the longevity of the pacemaker ... The high impedance of the lead ... electrode surface, combined with the elution ...
CapSure SP Novus endocardial, tined pacing leads provide the stable, low pacing thresholds and large sensing potentials already proven with CapSure SP steroid-eluting leads but within an even smaller...
The Vitatron Slimtine S lead series are endocardial leads with an excellent history of reliability and optimal handling en pacing performance....
Selute Picotip Atrial-J steroid eluting passive fixation lead...
Medicine Products: