Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome

September 2008
Current Medical Literature: Lysosomal Storage Disease;2008, Vol. 8 Issue 2, p68
Academic Journal
The article reports on the result of the study which assesses the effectiveness of enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) in treating patients with mucopolysaccharidosis type I (MPS I) or Hurler syndrome. The study was participated by seven patients who underwent HSCT for 11-14 weeks and combination of both treatments for 8 weeks. It shows that both treatments reduced pulmonary diseases, morbidity, and promotes normal α-L-iduronidase level.


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