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Title: Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation. Author: Pridjian G, Humbert J, Willis J, Shapira E. Journal: J Pediatr; 1994 Nov; 125(5 Pt 1):755-8. PubMed ID: 7965430. Abstract: At 8 months of age, before clinical neurologic deterioration, the younger of two sisters with metachromatic leukodystrophy received a transplant of bone marrow from her haploidentical, heterozygote mother. Compared with the course in the older, affected, untreated sibling, the onset of neurologic regression was delayed 1 year and progressed at a slower rate.[Abstract] [Full Text] [Related] [New Search]