Abstract
Donor bone marrow engraftment, which resulted in complete normalization of white cell β-galactosidase levels in a patient with presymptomatic juvenile onset GM1-gangliosidosis (McKusick 230600), did not improve long-term clinical outcome. © SSIEM and Springer 2005.
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CITATION STYLE
APA
Shield, J. P. H., Stone, J., & Steward, C. G. (2005). Bone marrow transplantation correcting β-galactosidase activity does not influence neurological outcome in juvenile GM1-gangliosidosis. Journal of Inherited Metabolic Disease, 28(5), 797–798. https://doi.org/10.1007/s10545-005-0089-7
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