title: Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy.
abstract: We compared substrate reduction in patients with lysosomal storage disorder treated with hematopoietic stem cell transplant and found that it was significantly reduced compared with patients treated with pharmacological enzyme replacement therapy. These data might support the wider application of hematopoietic stem cell transplant in the treatment of lysosomal storage disorders.
authors.affiliation: Department of Haematology and BMT, Royal Manchester Children's Hospital, Manchester, United Kingdom. robert.wynn@cmmc.nhs.uk
authors.forename: Robert F
authors.initials: RF
authors.lastname: Wynn
authors.forename: J Ed
authors.initials: JE
authors.lastname: Wraith
authors.forename: Jean
authors.initials: J
authors.lastname: Mercer
authors.forename: Anne
authors.initials: A
authors.lastname: O'Meara
authors.forename: Karen
authors.initials: K
authors.lastname: Tylee
authors.forename: Margaret
authors.initials: M
authors.lastname: Thornley
authors.forename: Heather J
authors.initials: HJ
authors.lastname: Church
authors.forename: Brian W
authors.initials: BW
authors.lastname: Bigger