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Amicus Therapeutics Presents Positive Results From Phase 1 Clinical,Studies of Plicera for Gaucher Disease

ent increase in GCase levels was observed in white blood cells from healthy volunteers receiving Plicera.

Phase 2 Clinical Trials of Plicera in Gaucher Disease

Based on the Phase 1 results, Amicus has initiated two Phase 2 clinical trials of Plicera for Gaucher disease. One is a 4-week study designed to evaluate the safety and pharmacodynamic effects of Plicera in Type I Gaucher patients who will discontinue enzyme replacement therapy for the duration of the study. The second is a 6-month study designed to evaluate the safety of Plicera and its effect on parameters that are commonly abnormal in Gaucher disease. This study will be conducted in Type I Gaucher patients who have never received enzyme replacement therapy. More information regarding these studies can be found at www.clinicaltrials.gov and www.amicustherapeutics.com.

About Gaucher Disease

Gaucher disease, the most commonly diagnosed lysosomal storage disorder, is caused by inherited genetic mutations in the GBA gene, which result in deficient activity of the enzyme acid beta-glucosidase, also known as glucocerebrosidase (GCase). Deficient GCase activity leads to lysosomal accumulation of glucocerebroside inside certain cells, which is believed to cause the various symptoms of Gaucher disease, including an enlarged liver and spleen, abnormally low levels of red blood cells and platelets and skeletal complications. In some cases there is significant impairment of the central nervous system. Gaucher disease affects an estimated 8,000 to 10,000 people worldwide. The U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation for the active ingredient in Plicera in the United States.

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics
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