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Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism

25 Oct 2019

Abstract excerpt

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 effort of several international centers, factors that predict successful patient and transplant outcomes have been...

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PMID: 31709204
DOI: 10.3389/fped.2019.00433

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