Abstract
Case Study: Krabbe disease (globoid cell leukodystrophy, Omim 245200) is a severe genetic degenerative brain disorder caused by the deficiency of the lysosomal enzyme β-galactocerebrosidase. The classical infantile form of the disease starts during the first year of life with dramatic loss of psychomotor functions and leads to early death. Therapy of this form with hematopoietic stem cell transplantation (HSCT) has been advocated for many years, but in spite of treatment early during the presymptomatic phase of the disease, has remained controversial. In contrast, a limited number of patients with late-onset forms of Krabbe disease receiving HSCT therapy have been reported; some of them suggesting HSCT as an effective treatment option.
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