Article
Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation.
The Journal of pediatrics - 1 Nov 1994
Pridjian G, Humbert J, Willis J, Shapira E
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...
Topics
- Bone Marrow Transplantation
- Brain
- Evoked Potentials, Auditory, Brain Stem
- Female
- Heterozygote
- Humans
- Infant
- Leukodystrophy, Metachromatic
- Magnetic Resonance Imaging
- Phenotype
- Remission Induction
- Time Factors