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Long-Term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Juvenile Metachromatic Leukodystrophy Compared with Nontransplanted Control Patients
JAMA Neurol 73:1133-1140, Groeschel, S.,et al, 2016
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Article Abstract
Among patients with juvenile MLD, patients who underwent HSCT had a better gross motor and language outcome and lower MRI severity scores compared with nontransplanted patients. Transplantation at a presymptomatic or early symptomatic stage of juvenile MLD is associated with a reasonable chance for disease stabilization.
 
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controversies in neurology
intellectual deterioration
metachromatic leukodystrophy
metachromatic leukodystrophy,juvenile
MRI,abnormal
neuropathy
prognosis
stem cell transplantation
treatment of neurologic disorder

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