FORMULATION AND METHOD FOR THE PREVENTION AND TREATMENT OF SKELETAL MANIFESTATION OF GAUCHER'S DISEASE

摘要

Summary Formulation and method for the prevention and treatment of skeletal manifestation of Gaucher's disease The invention relates to the prevention and treatment of skeletal manifestation of Gaucher's disease. It relates to a formulation for introducing glucocerebrosidase into the lysosomal compartment of macrophages and/or Gaucher cells in the bone marrow, the formulation comprising glucocerebrosidase encapsulated in erythrocytes. Said erythrocytes may have been subjected to a chemical treatment with an agent so as to promote the targeting of macrophages and/or Gaucher cells in the bone marrow. The treatment may be carried out with a bis(sulphosuccinimidyl) suberate (BS3) solution. The invention also describes drugs and methods for prevention and treatment. No figures.