Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation

William Krivit, Elsa Shapiro, William Kennedy, Meryl Lipton, Lawrence Lockman, Stephen Smith, C. Gail Summers, David A. Wenger, Michael Y. Tsai, Norma K.c. Ramsay, John H. Kersey, Jeffrey K. Yao, Edward Kaye

Research output: Contribution to journalArticlepeer-review

125 Scopus citations


METACHROMATIC leukodystrophy of the late infantile type, an autosomal recessive inherited disorder caused by a deficiency of arylsulfatase A activity, is characterized by progressive mental regression, loss of speech, quadriparesis, peripheral neuropathy, and death within a few years of onset.1 We describe a 10-year-old girl with metachromatic leukodystrophy in whom neurophysiologic function and sulfatide metabolism had improved after she received a bone marrow transplant five years before. These results may encourage the use of bone marrow transplantation in other patients with metachromatic leukodystrophy early in the course of the disease, when the degree of central nervous system impairment and the.

Original languageEnglish (US)
Pages (from-to)28-32
Number of pages5
JournalNew England Journal of Medicine
Issue number1
StatePublished - Jan 4 1990


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