Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy.
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.
Wynn
Robert F
RF
Department of Haematology and BMT, Royal Manchester Children's Hospital, Manchester, United Kingdom. robert.wynn@cmmc.nhs.uk
Wraith
J Ed
JE
Mercer
Jean
J
O'Meara
Anne
A
Tylee
Karen
K
Thornley
Margaret
M
Church
Heather J
HJ
Bigger
Brian W
BW