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Diseases

Genetic and Rare Diseases Information Center (GARD)

Sandhoff disease


Other Names for this Disease
  • Beta-hexosaminidase-beta-subunit deficiency
  • GM2 gangliosidosis, type 2
  • Total hexosaminidase deficiency
  • Hexosaminidase A and B deficiency Disease
  • Sandhoff-Jatzkewitz-Pilz disease
Related Diseases
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Treatment

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How might Sandhoff disease be treated?

Since there are no specific treatments for Sandhoff disease, management is symptomatic and supportive. Supportive treatment includes proper nutrition and hydration and keeping the airway open. Anticonvulsants may be used to control seizures. In recent studies, a small number of children have received an experimental treatment using transplants of stem cells from umbilical cord blood.  Although these limited trials have not yet produced a treatment or cure, scientists continue to study these and other investigational approaches.[1]  Additional information about investigational treatments for Sandhoff disease can be accessed through the National Tay-Sachs & Allied Diseases Association, Inc. web page for Sandhoff disease.
Last updated: 10/19/2011

References
  1. NINDS Sandhoff Disease Information Page. National Institute of Neurological Disorders and Stroke (NINDS). 2011; http://www.ninds.nih.gov/disorders/sandhoff/sandhoff.htm. Accessed 10/19/2011.


GARD Video Tutorial

  • Finding Treatment Information - A video developed by GARD Information Specialists that explains how you can find information about treatment for a rare disease.

    Finding Treatment Information

Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Sandhoff disease. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
Other Names for this Disease
  • Beta-hexosaminidase-beta-subunit deficiency
  • GM2 gangliosidosis, type 2
  • Total hexosaminidase deficiency
  • Hexosaminidase A and B deficiency Disease
  • Sandhoff-Jatzkewitz-Pilz disease
Related Diseases
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.