What are the Treatment Options for Lysosomal Storage Diseases?
The treatment of LSDs focuses on managing symptoms and slowing disease progression. Options include:
1. Enzyme Replacement Therapy (ERT): Administering synthetic enzymes to replace the deficient or malfunctioning ones. 2. Substrate Reduction Therapy (SRT): Reducing the production of the substrate that accumulates due to enzyme deficiency. 3. Hematopoietic Stem Cell Transplantation (HSCT): Replacing the patient's defective cells with healthy donor cells. 4. Symptomatic treatment: Addressing specific symptoms such as pain, seizures, or cardiovascular issues.
While these treatments can improve quality of life and slow progression, most LSDs are currently incurable, highlighting the need for ongoing research and development of new therapies.
