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Case Reports
. 1994 Nov;125(5 Pt 1):755-8.
doi: 10.1016/s0022-3476(94)70072-9.

Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation

G Pridjian  1 , J Humbert, J Willis, E Shapira
Affiliations
Case Reports

Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation

G Pridjian et al. J Pediatr. 1994 Nov.

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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