The approach of molecular medicine to the treatment of Gaucher disease

Abstract

This review study provides the most recent information on the therapies approved for the treatment of the disease. Material and methods. Narrative synthesis of specialized literature in databases: PubMed, Gene Cards, National Library of medicine, Google Scholar and Hinari of the years 2014 to 2024 were analyzed. Results. Enzyme replacement therapy is an effective tool in the treatment of Type 1 Gaucher disease. Treatment is done via infusion of imiglucerase. This is a synthetic substance called glucocerebrosidase.