• Overview
• Inheritance
• Symptoms
  • Liver and Spleen
  • Lungs
• Testing
• Treatment
• Resources

Treatment

At present, there is no specific treatment available for NPD. However, the following treatments are being studied for possible use in the future:

Bone marrow transplantation (BMT)
BMT has been accomplished in patients with type A NPD, but the procedure did not improve the neurological symptoms of the disease. Studies in animal models of type B NPD have shown a great improvement of symptoms when the procedure was performed early in life. Because of these results, there is hope that BMT may be a future treatment of type B NPD in humans.

Enzyme Replacement Therapy (Clinical Trials)
A clinical trial for an enzyme replacement therapy for type B NPD is currently in the planning stages.

Dietary Changes and Medication
Studies are being done to determine if lowering cholesterol, with changes in diet and/or the use of medications, benefits people with type C NPD.