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FDA Approval Expected Friday for Muscle

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Duke Health News 919-660-1306

DURHAM, N.C. -- U.S. commercial approval for Myozyme is expected this Friday, April 28. Myozyme has been developed for the treatment of Pompe disease, a debilitating, progressive and often fatal genetic muscular disorder.

A Duke University Medical Center team spent more than 20 years in research involving patients with Pompe disease and in developing the recombinant enzyme (Myozyme), a life-saving therapy for patients with this debilitating condition. Myozyme is the first treatment that offers hope to individuals with Pompe disease. The researchers previously showed that the enzyme was safe and effective in reversing symptoms of Pompe disease in quails affected with the disease. The enzyme therapy has been shown to improve heart and muscular functions and therefore prolong and enhance quality of life in infants born with Pompe disease, which usually claims the lives of affected children before they reach their first birthday.

The Food and Drug Administration is expected to act on the U.S. marketing application by April 28. Synpac (North Carolina) Inc., a drug development company in Research Triangle Park, and its parent company, Synpac Pharmaceuticals Ltd. of Cambois, England, began funding the Phase I/II trial at Duke until 2000 when Genzyme Corp. of Cambridge, Mass., took over funding.

Genzyme expects to complete enrollment soon in a clinical study involving patients with late-onset Pompe disease, which is intended to provide further support for Myozyme's use. The trial will continue throughout this year, and results will be available early next year.

The study is part of a large, collaborative effort at Duke University Medical Center to find an effective treatment for Pompe disease, which affects approximately one in 40,000 individuals. Children born with Pompe disease have a defect in the enzyme known as acid alpha-glucosidase (GAA), which normally processes glycogen and converts it to glucose that the body needs for energy. The defect causes glycogen to build up in muscle tissues throughout the body, including the heart, causing various muscles to degenerate. Pompe disease affects more than 5,000 people in the United States. If symptoms appear during infancy, the disease is usually fatal. It is usually less severe when symptoms first appear late in childhood, but even then, life expectancy is greatly decreased and the disease causes significant morbidity.

Duke University Medical Center has an on-campus satellite uplink for live or recorded television interviews. We are also equipped with ISDN connectivity for radio interviews.

For assistance in arranging interviews, please contact Becky Oskin at (919) 684-4966 or (919) 684-4148. After hours, please page the Duke Medical Center News Office at (919) 970-3671.

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