An online survey on burden of illness among families with post-stem cell transplant mucopolysaccharidosis type I children in the United States

Severe mucopolysaccharidosis type I (also known as Hurler syndrome) is a rare devasting recessive genetic disease caused by the deficiency of an enzyme. Hematopoietic stem cell transplant is the standard of ca...

Source: Orphanet Journal of Rare Diseases - February 18, 2019 Category: Internal Medicine Authors: Therese Conner, Francesca Cook, Vivian Fernandez, Karen Rascati and Vanessa Rangel-Miller Tags: Research Source Type: research