Navigation Links
AMT Receives EMEA Orphan Drug Designation for Duchenne Muscular Dystrophy
Date:10/12/2009

AMSTERDAM, October 13 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-080 for the treatment of Duchenne muscular dystrophy.

Orphan Drug Designation for Duchenne muscular dystrophy (DMD) entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-080 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

"We are proud to have received this Orphan Drug Designation for the treatment of Duchenne muscular dystrophy," said Jorn Aldag, Chief Executive Officer of AMT. "This designation is an important next step in the development of a treatment for this progressive and devastating disease."

About Duchenne Muscular Dystrophy

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

AMT is developing a gene therapy product for Duchenne muscluar dystrophy based on technology that results in "skipping" of the defective portion of the dystrophin gene resulti
'/>"/>

SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3

Related biology technology :

1. Reclast(R) Receives US FDA Approval as First and Only Once-Yearly Treatment for Women With Postmenopausal Osteoporosis
2. Ciphergen Biosystems Receives Noncompliance Letter From The Nasdaq Stock Market
3. Indevus Receives Approvable Letter From FDA for VALSTAR(R) for Bladder Cancer Therapy
4. Anesiva Receives FDA Approval for Zingo(TM), a New, Innovative Product to Reduce Pain Associated with Needle Insertion Procedures in Children
5. IsoTis Receives FDA Clearance for Accell Family of Products
6. Actavis Receives Approval of Fentanyl Transdermal System in the U.S.
7. AGA Medical Corporation Receives FDA and CE Mark Approvals for the AMPLATZER Vascular Plug II
8. IDM Pharma Receives Not Approvable Letter for Mifamurtide (L-MTP-PE) for the Treatment of Osteosarcoma
9. XTL Receives Staff Letter From NASDAQ
10. XTL Receives Staff Letter From NASDAQ; XTL may Transfer its ADR Listing to the NASDAQ Capital Market
11. Hemo-Stream(TM) Chronic Dialysis Catheter Receives FDA Clearance
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/27/2015)... , April 27, 2015 ... this release, please scroll to bottom . ... equities: Lexicon Pharmaceuticals Inc. (NASDAQ: LXRX ), ... Pharmaceuticals Corporation (NASDAQ: SNTA ), Spectrum Pharmaceuticals ... (NASDAQ: MDVN ). Free research report on ...
(Date:4/27/2015)... -- SynGen Inc., a company that focuses on the ... and progenitor cells from umbilical cord blood, bone marrow, ... has added three new members to its management team ... focus on product commercialization. Chuck Novak ... and Chief Financial Officer.  Chuck was most recently CFO ...
(Date:4/27/2015)... Massachusetts and HEIDELBERG , ... -- Addresses unmet need in treatment ... Health announced today it has been granted a patent in ... Receptor". This latest patent supports Molecular Health,s discovery that EPHB4 ... to EPO and promotes tumor growth. EPO stimulates the production ...
(Date:4/27/2015)... 27, 2015  MT Services LLC, a Lawsuit ... today that U.S. District Judge Joseph Goodwin ... conference status meeting in hopes to resolve more ... mesh manufacturers who are involved in multidistrict litigations ... been going on for some time, and Judge ...
Breaking Biology Technology:Market Updates on Biotech Stocks -- Lexicon Pharma, Idera Pharma, Synta Pharma, Spectrum Pharma, and Medivation 2Market Updates on Biotech Stocks -- Lexicon Pharma, Idera Pharma, Synta Pharma, Spectrum Pharma, and Medivation 3Market Updates on Biotech Stocks -- Lexicon Pharma, Idera Pharma, Synta Pharma, Spectrum Pharma, and Medivation 4Market Updates on Biotech Stocks -- Lexicon Pharma, Idera Pharma, Synta Pharma, Spectrum Pharma, and Medivation 5SynGen Inc. Expands Its Management Team 2Molecular Health Gains Patent for Novel Receptor Utilizing Computational Biomarker Discovery Technology 2Transvaginal Mesh Lawsuit News: Another Joint Conference Status Meeting Ordered by Judge to Resolve More than 70K TVM Lawsuits against Several Mesh Manufacturers, Says Lawsuitsettlementnews.com 2Transvaginal Mesh Lawsuit News: Another Joint Conference Status Meeting Ordered by Judge to Resolve More than 70K TVM Lawsuits against Several Mesh Manufacturers, Says Lawsuitsettlementnews.com 3
... towards building a microscopic device that can construct nano ... the peer-reviewed publication, International Journal of Nanomanufacturing from Inderscience ... , In his 1986 book, The Engines of Creation, ... nanotechnology - to create an assembler, a microscopic device, ...
... Year Period, DUBLIN, Calif., April 28 SuperGen ... the first quarter ended,March 31, 2008., Total revenues ... $4.4 million for the same prior year period. Total ... in royalty revenue, compared,with $3.8 million for the same ...
... 28 Sangamo,BioSciences, Inc. (Nasdaq: SGMO ), ... zinc finger DNA-binding proteins (ZFPs) for therapeutic gene,regulation ... G.,Wiggans to the company,s Board of Directors., ... Officer of,Connetics Corporation, a biotechnology company, from 1994, ...
Cached Biology Technology:Are nanobots on their way? 2Are nanobots on their way? 3SuperGen Reports 2008 First Quarter Financial Results 2SuperGen Reports 2008 First Quarter Financial Results 3SuperGen Reports 2008 First Quarter Financial Results 4SuperGen Reports 2008 First Quarter Financial Results 5SuperGen Reports 2008 First Quarter Financial Results 6SuperGen Reports 2008 First Quarter Financial Results 7SuperGen Reports 2008 First Quarter Financial Results 8SuperGen Reports 2008 First Quarter Financial Results 9Sangamo BioSciences Nominates Thomas G. Wiggans to Its Board of Directors 2Sangamo BioSciences Nominates Thomas G. Wiggans to Its Board of Directors 3Sangamo BioSciences Nominates Thomas G. Wiggans to Its Board of Directors 4
(Date:4/2/2015)... Fingerprint Cards (FPC) has received ... the distributor World Peace Industrial Group (WPI), part of ... the major part of the shipments will take place ... smartphone manufacturers in China . The ... communicated revenue guidance of + 1 000 MSEK for 2015.  ...
(Date:3/31/2015)... YORK , March 31, 2015   Guidepoint ... the launch of the Post-Surgical Pain Management TRACKER, part ... products tracking usage trends in the medical device and ... access to data including treatment volumes, market share, and ... the pain associated with joint surgery. The ...
(Date:3/24/2015)... 2015 Research and Markets ( http://www.researchandmarkets.com/research/kvdtcw/face_and_voice ) has ... Global Strategic Business Report" report to their offering. ... in US$ Thousands by the following Segments: Face Biometrics, and Voice ... Canada , Japan , ... East & Africa , and ...
Breaking Biology News(10 mins):Fingerprint Cards Receives Touch Fingerprint Sensor Order of SEK 110 Million 2Guidepoint Launches Post-Surgical Pain Management TRACKER 2Guidepoint Launches Post-Surgical Pain Management TRACKER 3Face and Voice Biometrics - Global Strategic Business Report 2015: Face Recognition Gains Prominence in Immigration Control 2Face and Voice Biometrics - Global Strategic Business Report 2015: Face Recognition Gains Prominence in Immigration Control 3Face and Voice Biometrics - Global Strategic Business Report 2015: Face Recognition Gains Prominence in Immigration Control 4Face and Voice Biometrics - Global Strategic Business Report 2015: Face Recognition Gains Prominence in Immigration Control 5Face and Voice Biometrics - Global Strategic Business Report 2015: Face Recognition Gains Prominence in Immigration Control 6
... in a way that had never been observed in ... Current Biology, a Cell Press publication. The discoverywhich marks ... to perceive circular polarized light may lend mantis shrimp ... into a new dimension of vision, said Justin Marshall ...
... minerals--mineral nanoparticles--in oceans and rivers, atmosphere and soils, ... new ways of understanding Earth,s workings. Our planet,s ... driven by the properties of these minerals. ... universities in a paper published in this week,s ...
... finches are becoming more common as pets, but the ... includes some of the most exotic creatures on the ... multi-billion dollar enterprise, but expansion of the industry sometimes ... that afflict these unusual animals. The new Manual ...
Cached Biology News:Mantis shrimp vision reveals new way that animals can see 2Mantis shrimp vision reveals new way that animals can see 3'Nanominerals' influence Earth systems from ocean to atmosphere to biosphere 2Veterinarians' guide to hedgehogs, chinchillas and chelonians.....oh, my! 2
Request Info...
Secure-Seal™ spacer, one well, 20 mm diameter, 0.12 mm deep *set of 100*...
Synphilin 1 Immunogen: Synthetic peptide corresponding to residues S(829) L E L N G E K D K D K G R T L Q R T(847) of human Synphilin 1. Storage: -20 C, Avoid Freeze/Thaw Cycles...
Request Info...
Biology Products: