Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation

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. (J P EDIATR 1994;125:755-8)