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Diseases

Genetic and Rare Diseases Information Center (GARD)

Mucopolysaccharidosis type I


Other Names for this Disease
  • MPS 1
  • Attenuated MPS I (subtype, includes Hurler-Scheie and Scheie syndrome)
  • Severe MPS I (subtype, also known as Hurler syndrome)
  • Hurler syndrome (subtype)
  • Hurler-Scheie syndrome (subtype)
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Treatment

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What treatment is available for mucopolysaccharidosis I (MPS I)?

The two main treatments for MPS I are enzyme replacement therapy (ERT) and bone marrow transplant. Both of these treatments work by replacing the missing IDUA enzyme. A drug called laronidase or Aldurazyme is the enzyme replacement therapy for MPS I. Treatment with laronidase can improve problems with breathing, growth, the bones, joints and heart. However, this treatment is not expected to treat problems with mental development because laronidase cannot cross the blood-brain barrier. A bone marrow transplant is another treatment option that provides the person with MPS I with cells that can produce the IDUA enyzme. A bone marrow transplant can stop the progression of neurological problems.[1]
Last updated: 7/15/2008

References
  1. Hurler's Syndrome and Transplant. National Marrow Donor Program Web site. http://www.marrow.org/PATIENT/Undrstnd_Disease_Treat/Lrn_about_Disease/Metabolic_Storage/Hurler_and_Tx/index.html. Accessed 7/15/2008.


GARD Video Tutorial

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Management Guidelines

  • GeneReviews provides current, expert-authored, peer-reviewed, full-text articles describing the application of genetic testing to the diagnosis, management, and genetic counseling of patients with specific inherited conditions.

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Mucopolysaccharidosis type I. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name laronidase
Trade Name
(Manufacturer Name)
Aldurazyme®
(BioMarin Pharmaceutical, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Treatment for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms
More Information about this product Drug Information Portal

Other Names for this Disease
  • MPS 1
  • Attenuated MPS I (subtype, includes Hurler-Scheie and Scheie syndrome)
  • Severe MPS I (subtype, also known as Hurler syndrome)
  • Hurler syndrome (subtype)
  • Hurler-Scheie syndrome (subtype)
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.