A whole-body perspective on Gaucher disease

Dr. Pramod K. Mistry, professor of pediatrics and internal medicine, explains that Gaucher disease does not only affect macrophage cell types. Related diseases such as cancer, Parkinson, and osteoporosis occur as a direct interaction with cells across the entire body. Currently approved options are successful at controlling Gaucher disease but do not focus on the entire body, he says, adding that the next step is to have an agent that can actively regulate lipid metabolism in all cells.