FDA approves new orphan drug to treat a form of Gaucher disease
date:May 04, 2012
at a dose of either 30 units per kilogram or 60 units/kg.


At both doses, Elelyso was effective in reducing spleen volume, the studys primary endpoint, from baseline by an average of 29 percent in patients receiving the 30 units/kg dose and by an average of 40 percent in patients receiving the 60 units/kg dose after nine months of treatment. Improvements in liver volume, blood platelet counts, and red blood cell (hemoglobin) levels also were observed.


In the other study, the efficacy of Elel
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