Long-term neurological outcome after hematopoietic stem cell transplant in juvenile Krabbe disease
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Background: Globoid cell leukodystrophy (GLD) is a progressive neurodegenerative disease caused by galactocerebrosidase deficiency. Juvenile phenotypes-onset between ages 3 and 16-account for up to 25% of cases. Hematopoietic ste... ...
