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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Mucopolysaccharidosis type IIIB


Other Names for this Disease

  • MPS 3B
  • MPS IIIB
  • Mucopoly-saccharidosis type 3B
  • N-acetyl-alpha-d-glucosaminidase deficiency
  • Sanfilippo syndrome B
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.

Your Question

My son has MPS IIIB, and I would like to know if there is any current research or clinical trials for this condition. This seems to be a rare disease that is also ‘rare’ in research and so it has been very difficult to obtain any treatment for my son. Are you able to help?

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

Are there any research studies or clinical trials for mucopolysaccharidosis type IIIB (MPS IIIB)?

The following resources provide information on research studies and clinical trials for MPS IIIB. We recommend that you contact these organizations directly, as they may be aware of additional research opportunities for MPS IIIB.

The Society for Mucopolysaccharide Diseases (UK) provides information on a research study for MPS IIIA and MPS IIIB. This project uses mouse models of MPS III to study the blood-brain barrier.

The National MPS Society suggests that clinical trials for the treatment of MPS IIIA are in the planning stages. Although this research is not specifically for MPS IIIB, the results could benefit individuals with MPS IIIB.

The Australian MPS Society provides additional information on current research for MPS III.

The U.S. National Institutes of Health, through the National Library of Medicine, developed ClinicalTrials.gov to provide patients, family members, and members of the public with current information on clinical research studies. Currently, one clinical trial is identified as involving MPS III (Sanfilippo syndrome), and the name of this study is Hematopoietic Stem Cell Transplantation (HSCT) for High Risk Inherited Inborn Errors. After you click on the study, review its "eligibility" criteria to determine its appropriateness. Use the study’s contact information to learn more. Check this site often for regular updates.

Orphanet lists clinical trials, research studies, and patient registries enrolling people with this condition. Click on Orphanet to view the list. 

GeneTests lists laboratories offering research genetic testing for this condition. Research genetic tests may be used to find disease-causing genes, learn how genes work, or aid in the understanding of a genetic disorder. In many cases test results are not shared with the patient or physician. Talk to your health care provider or a genetic professional to learn more about research testing for this condition.
Last updated: 2/10/2009
Other Names for this Disease
  • MPS 3B
  • MPS IIIB
  • Mucopoly-saccharidosis type 3B
  • N-acetyl-alpha-d-glucosaminidase deficiency
  • Sanfilippo syndrome B
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.