Navigation Links
PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy
Date:10/11/2007

ase 1 clinical trials, PTC124 was generally well tolerated, achieved target plasma concentrations that have been associated with activity in preclinical models and did not induce ribosomal read through of normal stop codons. PTC is currently conducting Phase 2 clinical trials of PTC124 in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD).

It is estimated that 10% of the cases of CF and 13% of the cases of DMD are due to nonsense mutations. PTC believes that PTC124 is potentially applicable to a broad range of other genetic disorders in which a nonsense mutation is the cause of the disease. The FDA has granted PTC124 Fast-Track designations and Orphan Drug designations for the treatment of CF and DMD due to nonsense mutations. PTC124 has also been granted orphan drug status for the treatment of CF and DMD by the European Commission. PTC124's development is supported by grants from the Muscular Dystrophy Association (MDA), Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), Parent Project Muscular Dystrophy (PPMD), FDA's Office of Orphan Products Development (OOPD) and by General Clinical Research Center grants from the National Center for Research Resources (NCRR).

About PTC Therapeutics, Inc.

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, small-molecule drugs that target post-transcriptional control processes. Post-transcriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTC's internally- discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology and infectious diseases. In addition, PTC has developed proprietary technologies and extensive knowledge of post-transcriptional control processes that it applies in its drug discovery and development activities, including the Gene Expression Modulation by Small-m
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2007 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related medicine technology :

1. Cell Therapeutics, Inc. Management to Discuss Todays Announcement of Interim Pixantrone Study Results
2. Halozyme Therapeutics Enhanze Technology Large Protein Molecule Therapeutic Clinical Trial Results Presented at the 34th Annual Meeting of the Controlled Release Society
3. CV Therapeutics Identifies Potential Anti-Diabetic Mechanism of Action for Ranexa
4. GTC Biotherapeutics to Webcast Corporate Presentation at the C.E. Unterberg, Towbin Emerging Growth Life Opportunities Conference
5. Fat Kills Cancer: Turning Stem Cells from Fat Tissue into Personalized, Cancer-Targeted Therapeutics
6. A 4-Week Therapy with Transition Therapeutics E1-I.N.T. Leads to Sustained Reductions in Blood Glucose Levels for 6 Months Post-treatment in Type 2 Diabetes Patients
7. Clinical Trial Suggests Bone Marrow Stem Cells Are Useful for Spinal Cord Injury; PrimeCell Therapeutics Provided Pre-Clinical Study
8. Pieris Progresses Development of its Proprietary Biotherapeutics Platform: Successful Demonstration of Dual Targeting With Duocalin(R) Technology
9. Opexa Therapeutics Reports Positive Top-line Data in Phase I/II Extension Trial with Tovaxin for Multiple Sclerosis
10. Orexigen Therapeutics to Present Data on Contrave Impact on Visceral Fat and Insulin Resistance at American Diabetes Association 67th Scientific Sessions in Chicago June 23
11. Allos Therapeutics Reports Results for Phase 3 Enrich Study of Efaproxyn in Women With Brain Metastases Originating From Breast Cancer
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/22/2014)... 21, 2014  Particle Sciences, Inc (PSI), the ... and manufactured a new innovative vaginal ring for ... Development arm of Exeltis Pharmaceuticals.  According to ... at Particle Sciences, "This vaginal ring was developed ... sciences and modeling expertise.  We are thrilled it ...
(Date:11/21/2014)... PARK, Md. , Nov. 21, 2014  The ... today will host the ceremonial groundbreaking of the new ... College Park. The new building will cultivate transformative new ... health.  Taking place at 10:00 a.m. on ... in the Paint Branch Parking Lot, adjacent to the ...
(Date:11/21/2014)... SARASOTA, Fla. , Nov. 21, 2014   ... ) announced that the Company is scheduled to present ... San Francisco, CA on January 12-14, ... PhD, Chairman and CEO of Rock Creek Pharmaceuticals, will ... small molecule cholinergic agonist which exhibits anti-inflammatory pharmacological characteristics. ...
Breaking Medicine Technology:Particle Sciences' Clinical Trial Supplies Group Manufactures Innovative Vaginal Ring 2University of Maryland Breaks Ground for A. James Clark Hall 2University of Maryland Breaks Ground for A. James Clark Hall 3University of Maryland Breaks Ground for A. James Clark Hall 4Rock Creek Pharmaceuticals to Present at the Biotech Showcase 2015 2Rock Creek Pharmaceuticals to Present at the Biotech Showcase 2015 3Rock Creek Pharmaceuticals to Present at the Biotech Showcase 2015 4
... Insmed Incorporated (Nasdaq CM: INSM), a biopharmaceutical company, today ... U.S. Food and Drug Administration (FDA) that it is ... 3 clinical trials for ARIKACE® (liposomal amikacin for inhalation) ... lung infections. Insmed has not yet received a response ...
... MAQUET Cardiovascular, a leading provider of cardiovascular ... three-year, dual-source contract with Premier Healthcare Alliance to ... membership.  The contract includes the CS300 product line, ... Catheter, SENSATION® 7Fr. IABP Catheter and LINEAR™ 7.5Fr. ...
Cached Medicine Technology:Insmed Incorporated Provides Regulatory Update 2Insmed Incorporated Provides Regulatory Update 3MAQUET Cardiovascular Awarded Three-Year Contract From Premier Healthcare Alliance for Intra-Aortic Balloon Pumps 2MAQUET Cardiovascular Awarded Three-Year Contract From Premier Healthcare Alliance for Intra-Aortic Balloon Pumps 3
(Date:11/24/2014)... 24, 2014 The Mesothelioma Applied ... annual International Symposium on Malignant Mesothelioma from March 2nd ... conference sessions will take place on the campus of ... including a community dinner and an awards ceremony, will ... will cover a range of topics about pleural and ...
(Date:11/24/2014)... (PRWEB) November 24, 2014 Olaparib functions ... polymerase (PARP) and is one of the first PARP ... to creating some types of melanoma, and are often ... also sometimes gives their malignancies a exclusive weaknesses, as ... to fix their DNA and allow them to proceed ...
(Date:11/24/2014)... The typical American diet includes foods ... Consistently consuming a diet high in calories and carbohydrates ... fat that typically accumulate around the internal organs exposing ... head in the form of love handles, beer bellies ... decision to rid themselves of the burden of embarrassing, ...
(Date:11/24/2014)... "My friend was killed when a vehicle ... street in his motorized wheelchair," said an inventor from ... up with this idea so that people in wheelchairs ... the Safety Lites to make a wheelchair highly visible, ... ensures that motorists can see the wheelchair. This helps ...
(Date:11/23/2014)... (PRWEB) November 24, 2014 Tbdress.com ... be available online on Nov. 28, 2014. The special offer ... , Tbdress.com is one of the world’s largest women’s ... styles and colors. People can enjoy great discounts on that ... can enjoy free delivery (for orders above $89). , People ...
Breaking Medicine News(10 mins):Health News:2015 International Symposium on Malignant Mesothelioma Will Take Place at the National Institutes of Health Campus 2Health News:2015 International Symposium on Malignant Mesothelioma Will Take Place at the National Institutes of Health Campus 3Health News:Olaparib Industry (CAS 763113-22-0) 2009-2019 Global and China Research Reports is Now Available at RnRMarketResearch.com 2Health News:Olaparib Industry (CAS 763113-22-0) 2009-2019 Global and China Research Reports is Now Available at RnRMarketResearch.com 3Health News:Olaparib Industry (CAS 763113-22-0) 2009-2019 Global and China Research Reports is Now Available at RnRMarketResearch.com 4Health News:Ketogenic Diet is the Perfect Solution for Diet Doc Patients Who Want to Learn How to Lose Weight over the Holidays 2Health News:Ketogenic Diet is the Perfect Solution for Diet Doc Patients Who Want to Learn How to Lose Weight over the Holidays 3Health News:Ketogenic Diet is the Perfect Solution for Diet Doc Patients Who Want to Learn How to Lose Weight over the Holidays 4Health News:Tbdress.com Special Friday Shoes Sales Online Now 2
... National Institute of Standards and Technology (NIST) researchers have ... of complex surfaces, which vary in a controlled fashion ... is so flexible that it can be applied to ... exploring the bonding of proteins to cell membranes. ...
... Indonesia's tourism industry, still shaky from terror bombings and ... May 27 earthquake devastated parts of this Javanese cultural ... ancient royal capital with its cultural history, temples and ... Bali for visits by overseas tourists. ,"We're ...
... was successfully by two crewmen in the early April. Followed ... the international space station within 6.5 hours on Thursday. // ... and 56 minutes. Except for a video camera to be ... a transport platform. ,After Russians have made their ...
... entered at the 20th annual Winnemucca Mule Races, Show & ... competition between clones each one won their qualifying heats, //but ... in U.S they lost to the traditionally bred mules. ... Gem, the world's first equine clone, had covered his 350-yard ...
... high school and college students are now co-authors in a ... 30 macrophages has been made possible by digging in the ... 2006 has a report of a research team lead by ... report is about the characterization and cataloguing of genomes of ...
... Clinical Nutrition says that the poor health and lifestyle of ... the society in its grip //. In a study of ... passed the habit to their sons and grandsons. ... Taiwan say that men who chewed betel nuts had children ...
Cached Medicine News:Health News:New Technique For Science Experiments 2Health News:Indonesian Tourism on Downward Trend After Java Quake 2
The Polaris-DX steerable diagnostic catheter is designed for comfort and ergonomic handling during mapping procedures....
... Atrial Isthmus Mapping. Biosense Webster offers ... catheters. The SANTORO Fixed Catheter is ... placement in the coronary sinus. The ... catheter's distal tip permit pacing in ...
... Torqr Quadripolar Braided Shaft ... The Medtronic CardioRhythm Torqr ... use in diagnostic electrophysiologic ... designed for recording intracardiac ...
The Explorer ST "soft tip" catheter has a proximal shaft with a soft tip. It is available in both a 5F and 6F shaft configuration....
Medicine Products: