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Diseases

Genetic and Rare Diseases Information Center (GARD)

Mucopolysaccharidosis type IIIA


Other Names for this Disease
  • Heparan sulfamidase deficiency
  • Heparan sulfate sulfatase deficiency
  • MPS 3A
  • MPS IIIA
  • Mucopoly-saccharidosis type 3A
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Treatment

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How might mucopolysaccharidosis type IIIA be treated?

Unfortunately, there is currently no cure or standard treatment for people with mucopolysaccharidosis type IIIA (MPS IIIA). Affected people and their families should consult with a medical genetics team for information about the diagnosis, counseling, up-to-date treatment options, and referrals to support groups and organizations. Treatment requires ongoing care with trained pediatric specialists in various medical fields. A consultation with a developmental pediatrician is especially helpful in addressing behavioral concerns and to assist parents and caregivers in establishing a sleep routine.

Currently, drug therapy is not part of the standard of care for MPS IIIA. Medications are used to relieve symptoms (such as anticonvulsants for seizures) and improve quality of life. Sedatives and melatonin have been used to improve the quality of sleep along with establishing a bedtime routine. Specific therapies such as bone marrow transplantation and enzyme replacement therapy (ERT) are currently not options for patients with MPS IIIA. Hematopoietic stem cell transplantation has shown mixed results and an unclear neurocognitive benefit. Recombinant enzymes for the deficiencies in MPS III are available, but trials in ERT have not been favorable in improving prognosis because the enzymes are not able to enter the central nervous system.[1] Changes to the diet do not prevent disease progression, but limiting milk, sugar, and dairy products has helped some people who have excessive mucus.[2]
Last updated: 7/21/2015

References
  1. Germaine L Defendi. Genetics of Mucopolysaccharidosis Type III. Medscape. March 21, 2014; http://emedicine.medscape.com/article/948540-treatment.
  2. Mucopolysaccharidoses Fact Sheet. NINDS. June 30, 2015; http://www.ninds.nih.gov/disorders/mucopolysaccharidoses/detail_mucopolysaccharidoses.htm.


GARD Video Tutorial

  • Finding Treatment Information - A video developed by GARD Information Specialists that explains how you can find information about treatment for a rare disease.

    Finding Treatment Information

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Mucopolysaccharidosis type IIIA. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
Other Names for this Disease
  • Heparan sulfamidase deficiency
  • Heparan sulfate sulfatase deficiency
  • MPS 3A
  • MPS IIIA
  • Mucopoly-saccharidosis type 3A
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.