Alglucerase for Gaucher's Disease: dose, costs, and benefits

Publication Authors:
Beutler E and Garber AM

Gaucher’s disease is the most prevalent glycolipid storage disorder.  A deficiency in the enzyme, glucocerebrosidase, causes the accumulation of glycolipid in a variety of organs.  In many cases, very possibly the majority, manifestations of the disease are so mild as to have a trivial effect on quality and length of life.  However, the disease can be devastating, resulting in massive enlargement of the liver and spleen, bone lesions, and in neurological manifestations in the rare type 2 and type 3 forms of the disease.  Alglucerase is a modified glucocerebrosidase preparation that has been found to be effective in the treatment of Gaucher’s disease.

(June 1994)

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