Gangliosidoses refers to a rare biochemical disorder that is characterised by a deficiency of an enzyme called beta-galactosidase A, which results in an accumulation of harmful chemicals in the body tissues as well as the central nervous system.
There is currently no treatment for Gangliosidosis that effectively treats it. Bone marrow transplantation has been found to be successful in patients who had infantile or juvenile Gangliosidosis, however, there was no long-term benefit reported. A possible treatment that has been advocated because of its success in treating other lysosomal storage disorders is the presymptomatic cord-blood hematopoietic stem-cell transplantation.
Symptomatic treatment for certain neurologic sequelae is available for treating the condition but it does not really alter the clinical course that has been advocated. Research in the area of enzyme replacement as well as gene therapy for Gangliosidosis has been going on for some time now but it has not advanced to human trials as yet.
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