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Amicus Therapeutics Announces Plan to Initiate Phase 1 Study of AT2220 for Pompe Disease
Date:9/30/2009

s of AT2220 on glycogen reduction in an appropriate transgenic mouse model of Pompe disease. Preliminary results of this study demonstrate consistent glycogen reduction across a range of different tissues, including heart, diaphragm and multiple skeletal muscles. Glycogen is the substrate that accumulates in the cells of patients with Pompe disease, which is believed to result in the clinical symptoms of the disease. The Company expects to present the results of this and other preclinical studies at appropriate scientific conferences in 2010.

Additionally, Amicus continues to be encouraged by the results of preclinical studies designed to evaluate the use of AT2220 in combination with enzyme replacement therapy (ERT). The Company expects to report additional data from these studies at scientific conferences in 2010.

About Pompe Disease

Pompe disease affects an estimated 5,000 to 10,000 individuals world-wide and is clinically heterogeneous in the age of onset, the extent of organ involvement, and the rate of progression. The early onset form of the disease is the most severe, progresses most rapidly, and is characterized by musculoskeletal, pulmonary, gastrointestinal, and cardiac symptoms that usually lead to death from cardio-respiratory failure between 1 and 2 years of age. The late onset form of the disease begins between childhood and adulthood and has a slower rate of progression that is characterized by musculoskeletal and pulmonary symptoms that usually lead to progressive muscle weakness and respiratory insufficiency. A high majority of people with Pompe disease have the late onset form. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in AT2220 in the United States.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as ph
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SOURCE Amicus Therapeutics
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