Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation

The long-term cognitive and functional outcomes of children with mucopolysaccharidosis type I (MPS-IH) post-hematopoietic cell transplant (HCT) are not well documented, and the role of genetic and treatment factors in these outcomes has yet to be defined. In this multi-site, international study, we...

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Vydáno v:JIMD Rep
Hlavní autoři: Kunin-Batson, A. S., Shapiro, E. G., Rudser, K. D., Lavery, C. A., Bjoraker, K. J., Jones, S. A., Wynn, R. F., Vellodi, A., Tolar, J., Orchard, P. J., Wraith, J. E.
Médium: Artigo
Jazyk:Inglês
Vydáno: Springer Berlin Heidelberg 2016
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5059216/
https://ncbi.nlm.nih.gov/pubmed/26825088
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2015_521
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