Bone marrow transplantation correcting beta-galactosidase activity does not influence neurological outcome in juvenile GM1-gangliosidosis.

J P H Shield, J Stone, C G Steward

Bristol Royal Hospital for Children, Bristol, UK. j.p.h.shield@bristol.ac.uk

Journal of inherited metabolic disease 2005

filter terms:

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.

Citation

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