Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation.
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.
Medical subject headings
- Bone Marrow Transplantation
- Leukodystrophy, Metachromatic