Currently, there is no cure for Niemann-Pick Disease, and treatment is primarily supportive. Management strategies aim to alleviate symptoms and improve the quality of life. Enzyme replacement therapy (ERT) has shown promise for Type B, where recombinant acid sphingomyelinase is administered to reduce lipid accumulation. For Type C, therapies such as miglustat (a substrate reduction therapy) and experimental treatments like gene therapy are being explored. Regular monitoring and supportive care, including physical therapy and nutritional support, are essential components of patient management.
