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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
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SOURCE Amsterdam Molecular Therapeutics B.V
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