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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.

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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
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SOURCE Amsterdam Molecular Therapeutics B.V
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