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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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.
Last updated: 7/15/2008
- 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.
- Finding Treatment Information - A video developed by GARD Information Specialists that explains how you can find information about treatment for a rare disease.
- 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.
- 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.
- The Orphan Disease Center: MPS I Pilot Grant Program presents a request for applications (RFA) to support research on the development of improved therapies for people with syndromes due to MPS I including Hurler, Hurler-Scheie and Scheie. All individuals holding a faculty-level appointment at an academic or non-profit institution are eligible to respond to this RFA. Grants will be awarded for an initial period of 1 to 2 years at $150,000 direct costs per year (up to 10% indirect costs allowable); funding for a second year is predicated by adequate progress during year 1 and availability of funding. All applicants must first submit a letter of Interest (LOI) to be reviewed for consideration of a full application submission. LOIs are due no later than Monday, February 29, 2016 at 5pm (EST).
(BioMarin Pharmaceutical, Inc.)
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|
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