FV 948. Diffusion Tensor Imaging Parameters in Metachromatic Leukodystrophy: Biomarkers for Disease Progress and Therapeutically Therapeutic Evaluation

Background: Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disease, which leads to progressive demyelination. Children with the early-onset late-infantile form suffer from rapid neurological progression, whereas children with the later onset juvenile form have a more slowly progressing disease process. Hematopoiesis stem-cell transplantation (HSCT) is currently the only clinically available therapy. In patients with the juvenile form, in particular, we were able to reveal a positive outcome, if the transplantation was performed at an early disease stage.