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

Abstract excerpt

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.

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Bone Marrow Transplantation
• Share to Brain
• Share to Heterozygote