Stem cell transplantation for adult-onset krabbe disease: report of a case.

Abstract

Krabbe disease is an autosomal recessive demyelinating lysosomal storage disorder caused by a deficiency of galactocerebrosidase. The adult-onset variant is very rare. Hematopoietic stem cell transplantation (HSCT) is reported to be successful in treating infants with Krabbe disease prior to the onset of symptoms, but there are no reported cases of its use… (More)
DOI: 10.1007/8904_2012_203

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@article{Sharp2013StemCT, title={Stem cell transplantation for adult-onset krabbe disease: report of a case.}, author={Madeleine E. Sharp and Cornelia Laule and Stephen H. Nantel and Burkhard M{\"a}dler and Ritu B Aul and Samuel Yip and Sandra M Sirrs}, journal={JIMD reports}, year={2013}, volume={10}, pages={57-9} }