Journal article
Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome
Sunita Bijarnia, Peter Shaw, Anne Vimpani, Robert Smith, Verity Pacey, Helen O'Grady, John Christodoulou, David 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.