NEW YORK, July 7, 2014 /PRNewswire/ -- Lysosomal storage disorders are a group of approximately 50 rare inherited metabolic disorders resulting from defects in lysosomal function. These severe, chronic genetic diseases have unmet medical needs, and include Fabry disease, Gaucher disease, Parkinson's disease, Pompe disease, and Tay-Sachs disease. The market for current drug therapies to address these disorders is estimated to exceed $3 billion.
Amicus Therapeutics, Inc. (NASDAQ: FOLD) is focused on discovering, developing, and commercializing small molecule, orally-administered pharmacological chaperones for the treatment of a variety of human genetic diseases. The company's focus is on treatments for rare and orphan human genetic diseases, such as lysosomal storage diseases (LSDs) and diseases involving neurodegeneration (the progressive loss of structure or function of neurons).
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