Currently, treatment options for Niemann-Pick Disease are limited and primarily focus on managing symptoms and improving quality of life. For type B, enzyme replacement therapy is being explored, while type C may benefit from medications that reduce cholesterol accumulation, such as miglustat. Gene therapy is also a promising area of research, aiming to correct the underlying genetic defects. Toxicologists play a crucial role in evaluating the safety and efficacy of these treatments, ensuring that they do not introduce new toxicological risks.
