Navigation Links
AMT Receives Orphan Drug Designation From the U.S. Food and Drug Administration Duchenne Muscular Dystrophy Gene Therapy
Date:9/24/2010

approval process. The designation provides the opportunity for AMT-080 to obtain market exclusivity for seven years from the drug's marketing approval date.

About Duchenne Muscular Dystrophy

DMD is a severe disease characterized by progressive muscle degeneration. It affects young children, almost exclusively boys, and leads to progressive paralysis and death in young adulthood. The disease is caused by mutations in the dystrophin gene, as a result of which the production of functional dystrophin protein, an important structural component within muscle tissue, is blocked. Currently, there is no treatment to prevent the fatal outcome of this disease. DMD affects one in 3,500 males, making it one of the most prevalent of muscular dystrophies.

About Amsterdam Molecular Therapeutics (AMT) Holding N.V.

AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using AAV as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. This safe and efficacious proprietary platform offers a unique manufacturing capability which can be applied to a large number of rare (orphan) diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products in LPLD, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson's Disease at different stages of research or development.

-------------------------------------------------------------------------

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of simila
'/>"/>

SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3 4 5 6 7 8

Related biology technology :

1. Professor Leroux From ETH Receives the Debiopharm Life Sciences Award 2010
2. NephRx Corp. Receives Orphan Drug Designation for NX001 for Prevention of Delayed Kidney Graft Function
3. MabVax Therapeutics Receives Small Business Innovation Research Program Award from the National Cancer Institute
4. NanoSmart™ Pharmaceuticals, Inc. Receives Patent Allowance for Tumor Targeting Platform Technology
5. Renhuang Receives AAA(1) Rating from Chinese Academy of International Trade & Economic Cooperation of Ministry of Commerce
6. Keck Graduate Institute Receives $3.6 Million NIH Grant
7. MacroGenics Receives NIH Grant Funding of $9.8M for Three Projects; Funding Further Advances DART Platform and Infectious Disease Portfolio
8. IIVS Receives First Mark Twain Ethical Science Award for Successes In Replacing Animals for Chemical and Product Safety Testing
9. Sirnaomics Receives Two SBIR Grants from NIH for Developing siRNA Therapeutics to Treat Glioblastoma and Influenza Infection
10. China Agritech, Inc. Receives $10 Million From Warrant Exercise by The Carlyle Group
11. Chiasma Receives Orphan Drug Designation From the FDA for Octreolin™ for the Oral Treatment of Acromegaly
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/31/2015)... DIEGO , Aug. 31, 2015 MabVax ... development company announces that the Company,s lead antibody, HuMab ... of five separate presentations during the upcoming World Molecular ... September 2-5, 2015. Researchers from the Department ... will present results on the use of MabVax,s lead ...
(Date:8/31/2015)... , Aug. 31, 2015  For people with ... brighten their day. The biotechnology company, located in the ... one of the best potential therapies yet for people ... entitled, " Correction of Down syndrome and Edwards syndrome ... journal DNA Research . Elixirgen plans to develop ...
(Date:8/31/2015)... 2015 FLX Bio, Inc., a biopharmaceutical company ... immunotherapies, announced today that it has appointed Brian ... William Ho , M.D., Ph.D., as Chief Medical ... team led by veterans of Flexus Biosciences, Inc., FLX,s ... Officer; Jay Powers , Ph.D., Vice President, Drug ...
(Date:8/31/2015)... , ... August 31, 2015 , ... There’s a new ... Minerva is the first commercially available new FDA approved system in the last 14 ... of 92%, the high Amenorrhea (zero bleeding) rate of 66%, the high Patient Satisfaction ...
Breaking Biology Technology:MabVax Therapeutics HuMab 5B1 Antibody Is Highlighted at Upcoming World Molecular Imaging Congress in Five Separate Presentations 2MabVax Therapeutics HuMab 5B1 Antibody Is Highlighted at Upcoming World Molecular Imaging Congress in Five Separate Presentations 3MabVax Therapeutics HuMab 5B1 Antibody Is Highlighted at Upcoming World Molecular Imaging Congress in Five Separate Presentations 4New Down syndrome therapy discovered 2FLX Bio Announces Senior Management Hires and Initiation of Phase 1 Trial of FLX925 2FLX Bio Announces Senior Management Hires and Initiation of Phase 1 Trial of FLX925 3FLX Bio Announces Senior Management Hires and Initiation of Phase 1 Trial of FLX925 4U.S. FDA Approves Minerva Endometrial Ablation System 2U.S. FDA Approves Minerva Endometrial Ablation System 3
... ABBOTT PARK, Ill. and SAN DIEGO , ... ) and Neurocrine Biosciences, Inc. (Nasdaq: NBIX ) ... agreement to develop and commercialize elagolix for the treatment ... gonadotropin-releasing hormone (GnRH) antagonist, which has recently completed a ...
... , ... The mother of a previously healthy 20 year old woman found dead four ... United States Supreme Court case where the Court has been asked to bar ... the Court were to bar such lawsuits, manufacturers would have a lessened incentive ...
... 11, 2010 Researchers at Texas A&M University College of ... with the successful delivery of a foal using oocytes from ... The delivery of the foal highlights Texas A&M,s long tradition ... great experience for the owner of the new foal., "I,ve ...
Cached Biology Technology:Abbott and Neurocrine Announce Global Agreement to Develop and Commercialize Elagolix for the Treatment of Endometriosis 2Abbott and Neurocrine Announce Global Agreement to Develop and Commercialize Elagolix for the Treatment of Endometriosis 3Abbott and Neurocrine Announce Global Agreement to Develop and Commercialize Elagolix for the Treatment of Endometriosis 4Abbott and Neurocrine Announce Global Agreement to Develop and Commercialize Elagolix for the Treatment of Endometriosis 5Supreme Court Amicus Brief Filed Opposing Lawsuit Protection for Manufacturers of Defectively Designed Vaccines 2Texas A&M veterinary researchers achieve cloning first 2Texas A&M veterinary researchers achieve cloning first 3
(Date:8/19/2015)...  VOXX International Corporation (Nasdaq: VOXX ) ... Definitive Agreement to purchase the assets, inclusive of ... EyeLock through an acquiring entity.  Upon consummation of ... in the acquiring entity. The closing of the ...  Expanding on its existing supply chain and distribution ...
(Date:8/12/2015)... 12, 2015 As the digital ... innovation and advanced biometrics technology is accelerating beyond expectations.  ... rely on using their credit cards, the digital currency, ... for payment services led by companies in the space ... Google, Inc. (NASDAQ: GOOG ), Apple Inc. (NASDAQ: ...
(Date:8/10/2015)... The latest 364 page report from business intelligence provider ... border security market . Visiongain assesses that this ... Now: Border security is hard to achieve and harder to ... you need to know about - and more importantly, you ... this will impact your company and the border security industry ...
Breaking Biology News(10 mins):VOXX International Corporation Signs Definitive Agreement To Acquire The Assets And A Controlling Interest Of Biometric Leader EyeLock 2VOXX International Corporation Signs Definitive Agreement To Acquire The Assets And A Controlling Interest Of Biometric Leader EyeLock 3VOXX International Corporation Signs Definitive Agreement To Acquire The Assets And A Controlling Interest Of Biometric Leader EyeLock 4VOXX International Corporation Signs Definitive Agreement To Acquire The Assets And A Controlling Interest Of Biometric Leader EyeLock 5Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 2Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 3Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 4Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 5Digital Wallet Devices & Mobile Payment Management Services Gain a Distinct Advantage in the Digital Payment Evolution as Demand Continues to Grow 6Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 2Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 3Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 4Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 5Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 6Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 7Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 8Border Security Market Report 2015-2025 - Growth in Biometrics, ICT, Manned and Unmanned Vehicles, and Perimeter Surveillance 9
... REDWOOD CITY, Calif., Dec. 2 DigitalPersona, Inc., a ... an agreement with IBM to deploy a new model ... 500 retail systems. The DigitalPersona U.are.U® 4500HD offering allows ... so that specific transactions can be linked to the ...
... of carbon about a nanometer in diameterhave been highly ... wires in molecular devices, or hydrogen storage components for ... at the National Institute of Standards and Technology (NIST) ... to the list: detection and destruction of an aggressive ...
... at the University of Southampton,s School of Ocean and ... (NOCS) has formally described four new species of king ... the new species in the Smithsonian Collections of the ... significance of the find, she said: "King crabs include ...
Cached Biology News:DigitalPersona Fingerprint Sensor Technology Ships on IBM SurePOS 500 Retail Systems 2Combining nanotubes and antibodies for breast cancer 'search and destroy' missions 2King crab family bigger than ever 2
Sterile glass 5 mm beads that make spreading bacterial and yeast cultures easy. Can be washed, autoclaved and reused....
Similar to proteasome (prosome, macropain) 26S subunit, ATPase, 1 Antigen: Recombinant Protein...
RABBIT ANTI AMPHOTERIN...
See product name for description....
Biology Products: