Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome.

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.

• Authors: Sunita Bijarnia, Peter Shaw, Anne Vimpani, Robert Smith, Verity Pacey, Helen O'Grady, John Christodoulou, David Sillence
• Journal: Journal of paediatrics and child health (J Paediatr Child Health) 2009 Jul-Aug Vol. 45 Issue 7-8 Pg. 469-72 ISSN: 1440-1754 [Electronic] Australia
• PMID: 19712183 (Publication Type: Case Reports, Journal Article)

Chemical References

• Iduronidase

Topics

• Child, Preschool
• Combined Modality Therapy
• Enzyme Replacement Therapy (methods)
• Female
• Follow-Up Studies
• Hematopoietic Stem Cell Transplantation
• Humans
• Iduronidase (deficiency, therapeutic use)
• Infant
• Mucopolysaccharidosis I (diagnosis, drug therapy, enzymology, surgery)
• Treatment Outcome