Journal article
Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome
S Bijarnia, P Shaw, A Vimpani, R Smith, V Pacey, H O'Grady, J Christodoulou, D Sillence
Journal of Paediatrics and Child Health | WILEY | Published : 2009
Abstract
We report the long-term follow-up of successful treatment of mucopolysaccharidosis type I H (MPS IH, Hurler syndrome) with combined enzyme replacement therapy and haematopoietic progenitor stem cell transplant. © 2009 Paediatrics and Child Health Division (Royal Australasian College of Physicians).