Published in Gene Therapy Weekly, August 31st, 2006
"MPS 1H is the severe form of mucopolysaccharidosis type 1 (MPS 1). HCT is the treatment of choice, but carries a high incidence of graft failure and morbidity.
"The use of ERT might improve the clinical signs and symptoms before HCT, resulting in less transplantation-related complications. Moreover, clearance of glycosaminoglycans (GAG's) from the bone marrow might improve engraftment," wrote J. Cox-Brinkman and colleagues, University of Utrecht.
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