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:9/30/2014)... 2014 China,s ... earthshaking changes. The country,s new leaders have recognized that ... reagents are far from perfect along with rapid population ... Chinese state council issued the latest "Regulations for the ... 2014, and it has come into force as June ...
(Date:9/29/2014)... and PARIS , Sept. 30, 2014 ... (EURONEXT: SAN and NYSE: SNY) today announced that a ... that blocks IL-4 and IL-13 signaling, met all primary ... with nasal polyps (CSwNP) who did not respond to ... potential of dupilumab for use in the treatment of ...
(Date:9/29/2014)... DUBLIN , Sept. 29, 2014  TNI ... biotechnology company pioneering the manufacturing both US and Internationally ... cancer, HIV/AIDS, opportunistic infections and autoimmune diseases in emerging ... Noreen Griffin The letter follows: Dear Shareholders, ... received a number of calls from shareholders and wants ...
Breaking Medicine Technology:Latest Chinese Guidebook for Application and Approval of Imported In-vitro Diagnostic Reagent Registration: From Regulations to Practices (2014 Edition) 2Latest Chinese Guidebook for Application and Approval of Imported In-vitro Diagnostic Reagent Registration: From Regulations to Practices (2014 Edition) 3Latest Chinese Guidebook for Application and Approval of Imported In-vitro Diagnostic Reagent Registration: From Regulations to Practices (2014 Edition) 4Latest Chinese Guidebook for Application and Approval of Imported In-vitro Diagnostic Reagent Registration: From Regulations to Practices (2014 Edition) 5Regeneron and Sanofi Announce Positive Phase 2 Top-line Dupilumab Results in Patients with Chronic Sinusitis with Nasal Polyps 2Regeneron and Sanofi Announce Positive Phase 2 Top-line Dupilumab Results in Patients with Chronic Sinusitis with Nasal Polyps 3Regeneron and Sanofi Announce Positive Phase 2 Top-line Dupilumab Results in Patients with Chronic Sinusitis with Nasal Polyps 4Regeneron and Sanofi Announce Positive Phase 2 Top-line Dupilumab Results in Patients with Chronic Sinusitis with Nasal Polyps 5Regeneron and Sanofi Announce Positive Phase 2 Top-line Dupilumab Results in Patients with Chronic Sinusitis with Nasal Polyps 6Regeneron and Sanofi Announce Positive Phase 2 Top-line Dupilumab Results in Patients with Chronic Sinusitis with Nasal Polyps 7TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 2TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 3TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 4TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 5TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 6TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 7TNI BioTech, Inc. Corporation's CEO Issues Letter to Shareholders Discussing Cytocom Dividend 8
... 9, 2011 The "Scientific Oktoberfest" will be ... distinguished researchers in the field of Chemical Biology. The so-called ... from the 15 th to the 16 th ... (LMU) München. At the two-day conference, 400 researchers ...
... Holdings, Inc., (NYSE: HRC ) today announced ... Item SA and Liko Care AG, the distribution companies ... Switzerland.  Hill-Rom, the global leader in patient support solutions, ... acquisition of Liko, A.B., a leading provider of patient ...
Cached Medicine Technology:"Scientific Oktoberfest" Draws the Worldwide Leading Researchers in the Field of Chemical Biology to Munich 2Hill-Rom Acquires Liko France and Switzerland 2Hill-Rom Acquires Liko France and Switzerland 3
(Date:9/30/2014)... Oxnard, CA (PRWEB) September 30, 2014 ... the Anacapa Dental Art Institute in Oxnard and Thousand ... Bangalore today and tomorrow to provide training on full ... previous dental work, placing implants, providing cosmetic dentistry, correcting ... Dr. Jivraj said. , Dr. Jivraj, who has traveled ...
(Date:9/30/2014)... If you were to create a list ... it say? Would you place a high value on charisma? ... create a leadership “wish list,” the truth is that just ... there different types of leaders. But if good leaders come ... programs that account for this kind of diversity? , ...
(Date:9/30/2014)... Lexington, KY (PRWEB) September 30, 2014 ... million people in the United States alone have sleep ... treated; and of those treated, many cannot tolerate their ... pressure supplied through a nasal mask) – which supports ... good night’s sleep has become especially valuable, and increasingly ...
(Date:9/30/2014)... is proud to announce the release of The ... by Frank Guglielmo and Sudhanshu Palsule ... their leadership coaching experience to introduce Social Leadership – a ... that leaders must adopt in order to thrive in today’s ... review of The Social Leader in Dialogue Reviews. , Author ...
(Date:9/30/2014)... IL (PRWEB) September 30, 2014 ... clash,” opened Bryan W. Brickner, “and it’s about ... Political Cannabinoid Science , new on the Bryan ... research from the National Institutes of Health (PubMed). ... regarding: de-homeostatic sleep disturbances, REM transitions, aging with ...
Breaking Medicine News(10 mins):Health News:World-Renowned Dental Implant Expert, International Instructor in India Speaking about Reconstructive Dentistry 2Health News:World-Renowned Dental Implant Expert, International Instructor in India Speaking about Reconstructive Dentistry 3Health News:World-Renowned Dental Implant Expert, International Instructor in India Speaking about Reconstructive Dentistry 4Health News:World-Renowned Dental Implant Expert, International Instructor in India Speaking about Reconstructive Dentistry 5Health News:Personality Plays a Key Role in Leadership Development 2Health News:Personality Plays a Key Role in Leadership Development 3Health News:Snoring: Robs People and Their Bed Partners of the Rest They Need to Face Daily Challenges 2Health News:Snoring: Robs People and Their Bed Partners of the Rest They Need to Face Daily Challenges 3Health News:The Social Leader: new Bibliomotion book redefines leadership for the Social Age 2Health News:The Social Leader: new Bibliomotion book redefines leadership for the Social Age 3Health News:The Social Leader: new Bibliomotion book redefines leadership for the Social Age 4Health News:The Social Leader: new Bibliomotion book redefines leadership for the Social Age 5Health News:The Social Leader: new Bibliomotion book redefines leadership for the Social Age 6Health News:Homeostasis: Publius’ Sleep Political Cannabinoid Science ~ New on the Bryan William Brickner Blog 2
... to have their blood sugar, blood pressure, and cholesterol ... a high-quality Medicare plan, researchers reported Tuesday. ... Medical Association, show that racial disparities are common throughout ... Amal N. Trivedi of Brown University in Providence, Rhode ...
... about personalized medicine enabled through genetic testing, slated to ... would not only involve forecasting the presence of a ... but will also enable early prevention strategies. ... cardiovascular diseases, but will unravel the genetic blue prints ...
... of folic acid every day could have better chance of getting ... form of vitamin B is used in our bodies to make ... body before she is pregnant, it can help prevent major birth ... study involving more than 18,000 women for the first time revealed ...
... Regular exercise combined with nicotine replacement therapy could help ... colleagues at the Otto Wagner Hospital// in Vienna enrolled ... nicotine-replacement therapy, reported the online edition of health magazine ... be quitters enrolled in an exercise programme., ,The ...
... to a recent study, women with a twin are at upto ... without one//. ,The study was conducted on over 800 ... premature ovarian failure (POF) at the age of 40 was 3-5 ... causes the ovaries to shut down ten years or earlier than ...
... and toddlers, reports a major study conducted on the side effects ... government proposed immunization for all kids of the age 6 months ... a sharp rise in the number of children getting vaccinated. This ... 2 years as often as the old people. ,This ...
Cached Medicine News:Health News:Medicare Marked by Widespread Racial disparities 2Health News:Folic Acid Supplements Could Help Conception 2
... system includes: Mx3005P instrument and custom ... five (5) filters. The standard filter ... HEX/JOE/VIC (535nm-555nm), ROX/Texas Red (585nm-610nm), Cy5 ... notebook computer, Mx3005P analysis software, Beacon ...
... SmartCycler System is a rapid, real-time thermal ... biological samples. By automating much of the ... individually programmable, it is the fastest, easiest ... on the market. The SmartCycler delivers highly ...
... SmartCycler System is a rapid, real-time thermal ... biological samples. By automating much of the ... individually programmable, it is the fastest, easiest ... on the market. The SmartCycler delivers highly ...
... The SmartCycler System is a rapid, real-time ... prepared biological samples. By automating much of ... site individually programmable, it is the fastest, ... now on the market. The SmartCycler delivers ...
Medicine Products: