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Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

Hematopoietic stem cell transplantation (HSCT) has been established as an effective therapy for selected inborn errors of metabolism. The success of HSCT in metabolic disease is best exemplified through the treatment of Hurler's syndrome, a lysosomal storage disease. Through the collaborative e...

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Foilsithe in:Front Pediatr
Main Authors: Tan, Emily Y., Boelens, Jaap Jan, Jones, Simon A., Wynn, Robert F.
Formáid: Artigo
Teanga:Inglês
Foilsithe: Frontiers Media S.A. 2019
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6824291/
https://ncbi.nlm.nih.gov/pubmed/31709204
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fped.2019.00433
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