Your browser does not support javascript:   Search for gard hereSearch for news-and-events here.

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)
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Research

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.
  • 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).

Patient Registry

  • The Lysosomal Disease Network is a team of doctors, nurses, research coordinators, and research labs throughout the U.S., working together to improve the lives of people with this condition through research. The Lysosomal Disease Network has a registry for patients who wish to be contacted about clinical research opportunities.
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.