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

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

Abstract Background 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 care in the United States, usually conducted before the child is 3 years...

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Bibliographic Details
Main Authors: Therese Conner, Francesca Cook, Vivian Fernandez, Karen Rascati, Vanessa Rangel-Miller
Format: Article
Language:English
Published: BMC 2019-02-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Mucopolysaccharidosis type I
Family burden of illness
Hurler syndrome
Online Access:http://link.springer.com/article/10.1186/s13023-019-1027-3

Internet

http://link.springer.com/article/10.1186/s13023-019-1027-3

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