Treatment of pathological bone conditions in patients with acid sphingomyelinase deficiency

摘要

The invention provides methods of using a human acid sphingomyelinase (e.g., olipudase alfa) in treating an abnormal bone condition in acid sphingomyelinase deficiency patients such as low bone density, high bone marrow burden, and other skeletal abnormalities presented in acid sphingomyelinase deficiency patients.