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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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Détails bibliographiques
Publié dans:Front Pediatr
Auteurs principaux: Tan, Emily Y., Boelens, Jaap Jan, Jones, Simon A., Wynn, Robert F.
Format: Artigo
Langue:Inglês
Publié: Frontiers Media S.A. 2019
Sujets:
Accès en ligne: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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