OBJECTIVES: The beneficial effects of macrophage-targeted glucocerebrosidase (Ceredase) in patients with Gaucher disease are well established. A minority of recipients develop transient nonneutralizin...
Efficient targeting of therapeutic reagents to tissues and cell types of interest is critical to achieving therapeutic efficacy and avoiding unwanted side effects due to offtarget uptake. To increase ...
The FDA recently approved Ceredase, a new treatment for Gaucher's disease, under the provisions of the Orphan Drug Act. Ceredase is unusually expensive, but there are no satisfactory alternative thera...