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Donor bone marrow engraftment, which resulted in complete normalization of white cell beta-galactosidase levels in a patient with presymptomatic juvenile onset GM1-gangliosidosis (McKusick 230600), did not improve long-term clinical outcome.


J P H Shield, J Stone, C G Steward. Bone marrow transplantation correcting beta-galactosidase activity does not influence neurological outcome in juvenile GM1-gangliosidosis. Journal of inherited metabolic disease. 2005;28(5):797-8

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PMID: 16151914

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