To date therapy has been entirely supportive. Hemato-poietic stem cell transplantation has been performed in fucosidase-deficient animals. Following successful engraftment, increased levels of fucosidase activity were found in leukocytes, plasma, and neural and visceral tissue. The enzyme reaches viscera and peripheral nerves rapidly via phagocytes, but it takes months to achieve substantial levels of enzyme activity in the CNS. Long-term engraftment from an early age reduced the severity and slowed the progression of clinical neurological disease in these animals; transplantation after the onset of clinical signs was not effective. Hematopoietic stem cell transplantation has been performed in a few patients and the results are promising in those undergoing the transplantation early. At present, it is still uncertain whether early treatment will completely prevent the clinical signs of disease.
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