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

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 of age,...

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Detalles Bibliográficos
Autores principales: Conner, Therese, Cook, Francesca, Fernandez, Vivian, Rascati, Karen, Rangel-Miller, Vanessa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6378738/
https://www.ncbi.nlm.nih.gov/pubmed/30777108
http://dx.doi.org/10.1186/s13023-019-1027-3

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