Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation.

Pridjian, G; Humbert, J; Willis, J; Shapira, E · J Pediatr · 1994

case_report · Level V

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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.

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