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EMA Starts Formal Review of Glybera(R) Dossier
Date:1/25/2010

esults from its follow-up and ongoing studies, in accordance with reporting regulations.

About the Disease

LPLD is a seriously debilitating, and potentially lethal, orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. This can result not only in pancreatitis, but also in difficult-to-treat diabetes, and is associated with significant morbidity and mortality.

About Amsterdam Molecular Therapeutics

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 LPL Deficiency, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson's Disease at different stages of research or development.

For information

AMT will be presenting at the BioCEO & Investor Conference, Waldorf-Astoria, New York City, at 9:30 am (EST) on Tuesday, February 9, 2010.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and ex
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SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2010 PR Newswire.
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