A 9-year-old boy is brought to the office by his mother for evaluation of diffuse bone pain in his left leg. He is below the 5th percentile for height and at the 10th percentile for weight. Physical examination shows tenderness to palpation of the left distal femur. There is hepatosplenomegaly. An x-ray of right femur shows generalized trabecular thinning and several osteolytic bone lesions. Laboratory results show anemia and thrombocytopenia. A bone marrow aspirate shows mononuclear cells filled with lipid that appear like wrinkled silk due to the accumulation of glycolipids within lysosomes. The patient is prescribed enzyme replacement therapy. Which of the following sets of pharmacologic properties best characterizes the administered medication?

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Eductional Objective: A deficiency of B-glucocerebrosidase is the cause of Gaucher disease, an autosomal recessive lysosomal storage disease. Gaucher disease manifests with hepatosplenomegaly, bone pathology, anemia, thrombocytopenia, and growth delays. Treatment is with recombinant glucocerebrosidase. Because enzymes are large proteins that cannot be orally absorbed, the replacement enzyme must be administered intravenously.  Entry into the cell occurs by endocytosis after the replacement enzyme binds to mannose 6-phosphate receptors on the cell surface.

A 9-Year-Old Boy Is Brought to the Office by His