Treatment at Mayo Clinic

By Mayo Clinic Staff


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No cure exists for Niemann-Pick disease. At this time, there is no effective treatment for type A. For type B, bone marrow transplantation is being studied as a possible treatment, along with enzyme replacement therapy and gene therapy. Miglustat (Zavesca) is approved for treatment of Niemann-Pick type C in Europe and in several countries but not in the U.S.

At Mayo Clinic, specialists work together to offer quality care to manage signs and symptoms, such as:

  • Sudden loss of muscle tone (cataplexy)
  • Seizures
  • Excessive muscle contractions (dystonia)
  • Sleep disturbances
  • Difficulty swallowing and eating
  • Recurrent pneumonia

Physical therapy is an important part of treatment to help maintain mobility as long as possible. Progressive symptoms of the disease require frequent medical follow-up.

April 02, 2014