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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Mucopolysaccharidosis type I


Other Names for this Disease
  • Alpha-L-Iduronidase deficiency
  • Attenuated MPS I (subtype)
  • Hurler syndrome (former subtype)
  • Hurler-Scheie syndrome (former subtype)
  • IDUA deficiency
More Names
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Treatment


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. Click on the link to view the article on this topic.

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