What are the Treatment Options for Lysosomal Storage Disorders?
Treatment strategies for LSDs are varied and may include:
- Enzyme Replacement Therapy (ERT): Administering the deficient enzyme to reduce substrate accumulation. - Substrate Reduction Therapy (SRT): Reducing the synthesis of the substrate that accumulates. - Bone Marrow Transplantation: In some cases, replacing the bone marrow can provide a source of healthy enzyme-producing cells. - Supportive Care: Symptomatic treatment to manage specific clinical manifestations.
