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FDA approves new orphan drug to treat a form of Gaucher disease
date:May 04, 2012
ase and should be administered by a health care professional every other week. Type 1 Gaucher disease is estimated to affect about 6,000 people in the United States.


Todays approval provides for a new enzyme replacement therapy for the select number of patients with Type 1 Gaucher disease, said Julie Beitz, M.D., director of the Office of Drug Evaluation III in FDAs Center for Drug Evaluation and Research. It also demonstrates FDAs commitment to developing treatments for rare diseases.


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