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Amicus Therapeutics Announces Positive Results From Phase 2 Clinical Trials of Amigal(TM) for Fabry Disease
Date:12/19/2007

ymptoms of Fabry disease, including pain, kidney failure and increased risk of heart attack and stroke. Amigal is designed to selectively bind to and stabilize a-GAL, which facilitates proper trafficking of the enzyme to the lysosomes, where it is needed to break down GL-3. In a previous Phase 1 study, Amigal was shown to be safe and well tolerated and to increase GLA levels in white blood cells in healthy human volunteers. Additionally, pre-clinical studies showed that treatment of Fabry transgenic mice with Amigal can increase GLA activity and significantly reduce GL-3 accumulation in heart, kidney, skin and plasma.

Fabry disease is estimated to affect approximately 5,000 to 10,000 people in the developed world, but recent evidence suggests that the disease may be significantly under diagnosed. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan designation for Amigal in the United States, and the European Commission has designated Amigal as an orphan medicinal product in the European Union.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. Amicus has completed Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of Gaucher disease. The Company has completed Phase 1 clinical trials of AT2220 for the treatment of Pompe disease.

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