Navigation Links
PTC Therapeutics Receives $2.6 Million in Government Grants
Date:6/15/2009

b>ABOUT ATALUREN (PTC124(TM))

Ataluren is an orally delivered, investigational new drug discovered by PTC Therapeutics. The drug is being developed as a new approach for the treatment of nonsense mutation genetic disorders. Nonsense mutations create a premature stop signal in the mRNA causing the ribosome to terminate translation before a full-length protein is generated. This causes the protein to be truncated and non-functioning. Ataluren is designed to allow the ribosome to continue translation of the mRNA, overriding the premature stop signal and leading to the formation of a functioning protein. Ataluren is a potential therapy for patients with nonsense mutation genetic disorders.

Ataluren has demonstrated proof of concept in Phase 2a clinical trials and is currently 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 Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), FDA's Office of Orphan Products Development, the Muscular Dystrophy Association, the Parent Project Muscular Dystrophy, and the
'/>"/>

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

Page: 1 2 3 4

Related biology technology :

1. Vicus Therapeutics to Present at the 234th American Chemical Society National Meeting in Boston, MA
2. DJO Incorporateds Pending Merger With ReAble Therapeutics Clears U.S. Antitrust Review
3. PeriCor Therapeutics Announces Closing of Licensing Agreement for Acadesine with Schering-Plough Corporation
4. Systems Medicine (SM), a Subsidiary of Cell Therapeutics, Inc. (CTI), Announces Execution of CRADA with NCI/NIH to Develop Potential Cancer Treatments Targeting the Insulin Growth Factor (IGF) Pathway
5. Amsterdam Molecular Therapeutics to Release H1 2007 Results on August 29, 2007
6. Nektar Therapeutics Appoints Tim Harkness as Chief Financial Officer
7. ExonHit Therapeutics: Allergan and ExonHits First Collaboration Compound to Begin Human Clinical Trials
8. Cell Therapeutics, Inc. Takes Corrective Actions in Response to Nasdaq Determination
9. Amsterdam Molecular Therapeutics Reports Half Year Results 2007
10. Nektar Therapeutics Appoints Lutz Lingnau as New Board Member
11. Anesiva Grants Specific-Use License of Its Needle-Free Drug Delivery Technology to Particle Therapeutics for Diabetes Drug
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/25/2014)... A new study by researchers from the University ... tiny nanosystems function, unlocking the potential to create ... study, which has been published in the prestigious ... a novel laser technique to examine in rich ... a small cluster containing an acetylene molecule and ...
(Date:7/25/2014)... WABC Radio show out of New York City, ... will be hosting California-based Vet-Stem, Inc.’s Founder and CEO, Robert ... pets. Dr. Harman first visited the show in November ... pets suffering from osteoarthritis and other degenerative diseases, as well ... , Young has invited Dr. Harman back to dial down ...
(Date:7/25/2014)... 25, 2014  Galmed Pharmaceuticals Ltd. (Nasdaq: ... the development and commercialization of a once-daily, oral therapy ... today announced financial results for the period ended June ... , Reported cash and cash equivalents totaling $37.4 ... December 31, 2013.  , Reported a net loss ...
(Date:7/25/2014)... Biotech Ltd. (Nasdaq: SVA ), a leading provider ... announced that it will release its unaudited financial results for ... on Thursday, August 14, 2014 EDT. The Company will host ... August 15, 2014, at 8:00 a.m. EDT (Friday, August 15, ... Company,s financial results and provide an update on recent corporate ...
Breaking Biology Technology:Breakthrough laser experiment reveals liquid-like motion of atoms in an ultra-cold cluster 2Breakthrough laser experiment reveals liquid-like motion of atoms in an ultra-cold cluster 3Robert J. Harman, DVM, Founder and CEO of Vet-Stem, Inc. to Join New York Radio Host Lorry Young in Upcoming Episode of A “Paws” For Your Pet 2Robert J. Harman, DVM, Founder and CEO of Vet-Stem, Inc. to Join New York Radio Host Lorry Young in Upcoming Episode of A “Paws” For Your Pet 3Galmed Pharmaceuticals Reports First Half 2014 Financial Results 2Galmed Pharmaceuticals Reports First Half 2014 Financial Results 3Galmed Pharmaceuticals Reports First Half 2014 Financial Results 4Galmed Pharmaceuticals Reports First Half 2014 Financial Results 5Galmed Pharmaceuticals Reports First Half 2014 Financial Results 6Sinovac to Host Conference Call to Report Second Quarter 2014 Unaudited Financial Results 2
... Researchers Comprise One of the Main User Groups of febit,s ... Simplified and Automated Microarray Technology, MEDFORD, Mass., ... it will introduce the Geniom RT Analyzer(R) to,the U.S. market ... in Boston., RNA-researchers, one of the main user groups ...
... SANTA CLARA, Calif., April 22 /PRNewswire/,-- Agendia BV, ... of molecular,diagnostics, and Agilent Technologies, Inc. (NYSE: ... to collaborate to develop new in-vitro,diagnostic tests. In ... Agendia products will continue to be supplied on ...
... SUPG ), a pharmaceutical company dedicated to the discovery, ... malignancies, invites all interested parties to listen and,view a ... at 9:30 a.m. ET,(6:30 a.m. PT) to approximately 12:00 ... Investors and interested parties may access the webcast ...
Cached Biology Technology:febit to Introduce Geniom RT Analyzer(R) to U.S. Market at RNAi World Congress 2febit to Introduce Geniom RT Analyzer(R) to U.S. Market at RNAi World Congress 3Agendia and Agilent Announce Plans to Jointly Develop New Diagnostic Tests, Extend Supply Agreement 2Agendia and Agilent Announce Plans to Jointly Develop New Diagnostic Tests, Extend Supply Agreement 3
(Date:7/25/2014)... a pleiotropic factor characterized by the existence of ... that confer to the protein deeply different characteristics. ... myelination occurring during development and the different phases ... degeneration, axon regrowth, remyelination and target reinnervation, Researchers ... that the soluble NRG1 upregulation observed in Schwann ...
(Date:7/25/2014)... of a molecular testing panel developed at UPMC ... initial surgery for patients with thyroid nodules and ... Cancer Institute (UPCI), partner with UPMC CancerCenter., The ... and other diagnostic testing agencies, improved the chances ... 30 percent, according to the study published this ...
(Date:7/24/2014)... -- Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX) (the "Company") announced the ... Phase 3 study of Zerenex (ferric citrate), the Company,s ... of hyperphosphatemia in patients with end-stage renal disease (ESRD) ... delivery with FErric CiTrate in EsrD) was published online ... of Nephrology ( JASN ). , ...
Breaking Biology News(10 mins):Test increases odds of correct surgery for thyroid cancer patients 2Test increases odds of correct surgery for thyroid cancer patients 3Zerenex™ (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 4
... , AMIA, the association for professionals in biomedical and ... the National Coordinator on Health Information Technology (DHHS/ ONCHIT), ... meaningful use of electronic health records (EHRs) over the ... Policy Committee (HITPC), on the continuation of support for ...
... When patients receive a bone marrow transplant, they are getting ... are needed when a patient is low on red blood ... be caused by cancer or even cancer treatments such as ... transplant might not succeed because the transplanted stem cells don,t ...
... Spanish . Fernando Rendo, biologist at the ... with the intention of completing the genealogical tree ... Country Autonomous Community (CAPVEAE) and Navarre. Concretely, he opted for ... (a kind of variation in the DNA sequence) that can ...
Cached Biology News:AMIA on meaningful use: Invest in people and technology 2How long do stem cells live? 2Basque thesis investigates wide diversity within certain ovine, bovine and equine breeds 2Basque thesis investigates wide diversity within certain ovine, bovine and equine breeds 3
MitoScreen (JC-1) Kit , 100 tests Consult technical datasheet for details....
Large-fragment, single-stranded carrier DNA of the highest quality, perfectly suited for all types of yeast transformation....
... , Customer Peptide Synthesis , 20 Amino Acid length ... 20 mg will return to customer for future usage conjugation ... , Maintenance 102 days/each , ... x5 /each , Test bleed (app. 5 ml) ...
Request Info...
Biology Products: