Metachromatic Leukodystrophy

Treatment

Doctors who have training in nervous system conditions (neurology) and other specialties coordinate your treatment. Doctors work with you to determine the most appropriate treatment for your needs.

Metachromatic leukodystrophy can't be cured, and few treatment options are available. Your doctors will work with you to help manage your condition and your symptoms and potentially improve your quality of life. You may have access to clinical trials.

Your doctor may help you manage your condition using several treatments:

• Medications. Medications may reduce your symptoms and relieve your pain.
• Bone marrow transplant. Bone marrow transplant sometimes has slowed the progression of metachromatic leukodystrophy.
• Physical, occupational and speech therapy. You may have physical therapy to move your muscles and joints, to keep joints flexible and maintain your range of motion as much as possible. You may have occupational and speech therapy to improve your quality of life.
• Nutritional assistance. You and your family may work with a nutrition specialist (dietitian) to determine how to provide your nutrition, as it may become difficult to swallow food or liquid. You may need assistive feeding devices as your condition progresses.
• Follow-up care. Doctors will coordinate your follow-up care with your primary doctor. In follow-up care, doctors will monitor you for changes in movement, swallowing, communication, behavior and other functions. Mayo Clinic staff offers various resources, education and information for your family and can help connect you to support groups. You and your family also may work with a genetic counselor.