What are the Treatment Options for Lysosomal Storage Disorders?
While there is no cure for most LSDs, several treatment options can help manage symptoms and improve quality of life:
- Enzyme Replacement Therapy (ERT): Administers synthetic enzymes to replace the deficient ones. - Substrate Reduction Therapy (SRT): Reduces the production of the substrates that accumulate in cells. - Hematopoietic Stem Cell Transplantation (HSCT): Can provide a long-term source of functional enzymes. - Supportive Care: Includes physical therapy, nutritional support, and respiratory care to manage symptoms.
