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D. Kidd, MD; J. Nelson, MD; F. Jones, MRCPath; H. Dusoir; I. Wallace, MSc; S. McKinstry, FRCR; V. Patterson, FRCP

Arch Neurol. 1998;55:98-99.

We describe a 16-year-old boy with juvenile metachromatic leukodystrophy who was treated with bone marrow transplantation. Follow-up over 8 years showed no increase in symptoms, no progression of neurological signs, and no neuropsychological deterioration. We conclude that bone marrow transplantation may increase brain levels of arylsulfatase A enough to prevent deterioration in patients with juvenile metachromatic leukodystrophy.

From the Departments of Neurology (Drs Kidd and Patterson), Medical Genetics (Dr Nelson), Hematology (Dr Jones), Neuropsychology (Ms Dusoir), Neuropathology (Mr Wallace), and Neuroradiology (Dr McKinstry), Royal Victoria Hospital, Belfast, Northern Ireland. Dr Kidd is now with the National Hospital for Neurology and Neurosurgery, London, England, and Dr Nelson is with Genetics Services of Western Australia, King Edward Memorial Hospital, Perth.

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ABSTRACT