Other Names for this Disease
- Beta galactosidase 1 deficiency
- GLB 1 deficiency
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Bone marrow transplantation was reportedly successful in an individual with infantile/juvenile GM1 gangliosidosis; however, no long-term benefit was reported. Presymptomatic cord-blood hematopoietic stem-cell transplantation has been advocated by some as a possible treatment due to its success in other lysosomal storage disorders. Active research in the areas of enzyme replacement and gene therapy for the condition is ongoing but has not yet advanced to human trials.
Neurologic and orthopedic sequelae may prevent adequate physical activity, but affected individuals may benefit from physical and occupational therapy.
- David H. Tegay. GM1 Gangliosidosis. Medscape Reference. March 29, 2012; http://emedicine.medscape.com/article/951637-overview. Accessed 8/6/2012.
- Tegay D. GM1 Gangliosidosis. eMedicine. March 29, 2012; http://emedicine.medscape.com/article/951637-overview. Accessed 4/3/2012.
- GM1 Gangliosidosis - Infantile. Hide & Seek Foundation for Lysosomal Disease Research. http://www.hideandseek.org/Diseases.html. Accessed 4/3/2012.
- NINDS Gangliosidoses Information Page. National Institute of Neurological Disorders and Stroke (NINDS). October 2011; http://www.ninds.nih.gov/disorders/gangliosidoses/Gangliosidoses.htm. Accessed 4/3/2012.
Clinical Trials & Research for this Disease
- ClinicalTrials.gov lists trials that are studying or have studied GM1 gangliosidosis. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.