Navigation Links
PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy
Date:2/3/2009

in a registration-directed pivotal study with the goal of demonstrating that allowing expression of a critical missing protein will safely provide clinical benefits for patients with nonsense mutation genetic disorders. Ataluren has been generally well tolerated across all clinical studies to date.

The FDA and the European Commission have granted PTC124 (ataluren) orphan drug status for the treatment of DMD and cystic fibrosis due to nonsense mutations. The FDA has also granted PTC124 (ataluren) Subpart E designation for expedited development, evaluation, and marketing.

PTC has an exclusive collaboration with Genzyme Corporation for the development and commercialization of ataluren. PTC Therapeutics will market ataluren in the United States and Canada, while Genzyme will commercialize the product in other regions of the world. The development of ataluren has also been supported by grants from the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, FDA's Office of Orphan Products Development, the National Center for Research Resources and the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), which recently expanded support of ataluren to include funding up to $25 million.

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. PTC has extensive knowledge of post-transcriptional control processes and has developed proprietary technologies that it applies in its drug d
'/>"/>

SOURCE PTC Therapeutics, Inc.
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3 4 5

Related medicine technology :

1. DNA Therapeutics Closes 2.2 Million EUR Series A Financing Round
2. Synvista Therapeutics Announces Termination of Clinical Trials of Alagebrium and SYI-2074 and Provides Business Update
3. Duska Therapeutics Completes Corporate Name Change to Cordex Pharma
4. Phico Therapeutics Receives GBP1 Million Award From the Wellcome Trust to Take Anti-MRSA Nasal Product Through Early Clinical Trials
5. Pixantrone Pre-NDA Communication from FDA Provides Cell Therapeutics Basis to Begin Rolling NDA Submission
6. Cephalon Signs Option Agreement to Acquire Ception Therapeutics
7. Amicus Therapeutics Announces Positive Progress of Three Lead Clinical Programs and General Outlook for 2009
8. Cara Therapeutics Initiates Phase II Proof-Of-Concept Trial of Novel Analgesic in Acute Post-Operative Pain
9. ExonHit Therapeutics Expands Strategic Collaboration With Allergan
10. Synvista Therapeutics Announces Published Data Demonstrating Elevation of Carboxymethyl-Lysine (CML) Is Associated with Decline in Renal Function and Arterial Stiffness in Older Adults
11. Synvista Therapeutics Announces First Dosing of SYI-2074 in Clinical Trial for Psoriasis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/1/2015)... DUBLIN , Aug. 31, 2015 ... the addition of the "Introduction to Veterinary ... to their offering. This course ... a good introduction to those concerned with Veterinary ... experienced personnel who reqire a better understanding of ...
(Date:9/1/2015)... Egalet Corporation (Nasdaq: EGLT ) ... on discovering, developing, and commercializing innovative pain treatments, ... data on SPRIX ® (ketorolac tromethamine) Nasal ... candidate, and Egalet-002, an abuse-deterrent, extended-release, oral oxycodone ... to be held September 8 to 12 in ...
(Date:9/1/2015)... , Sept. 1, 2015  Based on ... management market, Frost & Sullivan recognizes Pie ... Frost & Sullivan Award for Technology Leadership. ... has designed innovative and easy-to-use imaging solutions—its ... and reproducible quantitative results for cardiovascular diagnosis ...
Breaking Medicine Technology:Introduction to Veterinary Pharmacovigilance Seminar (London, UK - September 23-24) 2Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 2Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 3Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 4Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 5Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 6Frost & Sullivan Applauds Pie Medical Imaging for Highly Innovative Quantitative Software for Cardiovascular Diagnosis 2Frost & Sullivan Applauds Pie Medical Imaging for Highly Innovative Quantitative Software for Cardiovascular Diagnosis 3Frost & Sullivan Applauds Pie Medical Imaging for Highly Innovative Quantitative Software for Cardiovascular Diagnosis 4Frost & Sullivan Applauds Pie Medical Imaging for Highly Innovative Quantitative Software for Cardiovascular Diagnosis 5
... Caliper Life Sciences, Inc. (Nasdaq: ... announced a co-marketing agreement which will leverage Covaris, ... microfluidics technologies to develop automated workflows for next ... suite of solutions for library construction, sample analysis, ...
... Pharmaceuticals, Inc. (Nasdaq: AVNR ) today announced that company ... in March. Citi 2011 Global Healthcare Conference ... Tuesday March 1, 2011 Presentation time: 8:00am ET ... Location: Boston Marriott, Boston, MA Presentation date: Tuesday March ...
Cached Medicine Technology:Caliper and Covaris Establish Co-Marketing Agreement to Simplify Next Generation Sequencing 2Caliper and Covaris Establish Co-Marketing Agreement to Simplify Next Generation Sequencing 3
(Date:9/1/2015)... ... September 01, 2015 , ... e-Spirit Inc., a leading supplier ... FirstSpirit Content Management System with new capabilities and enhancements designed to support the ... will be well on their way to achieving digital transformation with less time ...
(Date:9/1/2015)... ... 2015 , ... The FSH Society , the Massachusetts-based charity that has ... be the beneficiary of a collection of fundraising events this fall. From Boston to ... FSH Society and further FSHD research. , The list of 2015 fall ...
(Date:9/1/2015)... ... September 01, 2015 , ... METTLER ... Instruments in accordance with GMP requirements“ , featuring Ed Szczesny (Quality Assurance, Rhodes ... at 11:00 EDT (15:00 GMT). , Calibration, qualification, and the appropriate level ...
(Date:9/1/2015)... (PRWEB) , ... September 01, 2015 , ... ... date, more than 1,500 investor-families have received permanent green cards (I-829 petition approval) ... , The I-829 petition is the final step of the EB-5 visa process ...
(Date:9/1/2015)... ... 2015 , ... Situations requiring fast, easy-to-understand business and personal ... time for everyone involved. These occasions highlight the value of a professional translation ... has real people available to handle any translation assignment at any time of ...
Breaking Medicine News(10 mins):Health News:e-Spirit boosts Corporate Content Cloud initiative with release of FirstSpirit 5.2 2Health News:e-Spirit boosts Corporate Content Cloud initiative with release of FirstSpirit 5.2 3Health News:FSH Society Beneficiary of Variety of Fall Fundraising Events 2Health News:FSH Society Beneficiary of Variety of Fall Fundraising Events 3Health News:Live Webinar from Pharmaceutical Technology and METTLER TOLEDO on Calibration and Qualification of Laboratory Instruments 2Health News:CanAm Enterprises Reaches Another Milestone with More Than 1,500 I-829 Petition Approvals 2Health News:Universal Translation Services Provides Quality Translations 24 Hours a Day Including Weekends 2Health News:Universal Translation Services Provides Quality Translations 24 Hours a Day Including Weekends 3
... leukemia cells are thought to be inhabited by a rather ... attached// together in a pattern which is exactly the opposite ... is far removed from the theory prescribed in the molecular ... shift in the original thinking that relied on the information ...
... University of Kentucky being claims to one day change the ... to open up blocked arteries that feed the heart. ... author of the study that is to appear in the ... claimed that the drug ‘Enoxaparin’, that has been studied has ...
... 8 that treatment for a broad range of food allergies ... found// treatments by altering the proteins in the food that ... system. ,"Therapies for food allergy will be on ... Ree, of the University of Amsterdam, who is leading work ...
... Morbid obesity may have a deleterious effect on young, ... that obesity in toddlers is linked to lower IQ ... obesity with metabolic problems like diabetes, high cholesterol and ... Driscoll, a professor of pediatric genetics at the University ...
... in New Zealand are failing to get regular mammograms ... reports.// ,Breast Cancer Aotearoa Coalition Deputy Chairwoman ... the eligible women have taken the offer for free ... the increased waiting times are responsible for this. Mammograms ...
... Christchurch surgeon has put forth the idea that it would ... that had earlier been opposed and led to the// removal ... ,The Canterbury District Health Board had removed the patients from ... comply with the Health ministry policy. Nearly Seventy-four of the ...
Cached Medicine News:Health News:Enoxaparin To Become The Standard Treatment During Procedures For Blocked Arteries 2Health News:Cure for food allergies is less than a decade away 2Health News:Obesity Takes its Toll on IQ of Toddlers 2
... quick and easy automated refill system ... space on your laboratory bench and ... easy-to-use, one-hand system, you only need ... of Diamond Tips. All sizes of ...
... TowerPack is an innovative, quick ... Diamond Tips. Use 60% less space ... less plastic. With this amazingly easy-to-use, ... box for the whole range of ...
10 L, Racked, 96 tips/rack Extended length pipette tips recommended for 5 and 10 L pipettes....
This pipette tip facilitates the loading of samples onto polyacrylamide gels for DNA sequencing. The Geloader Tip has a 15 mm capillary with a defined diameter of less than 0.3 mm. The flexibility of...
Medicine Products: