Hematopoietic stem cell transplantation in inborn errors of metabolism

HIGHLIGHTS

  • who: Robert F. Wynn from the Faculty of Health and Medical Sciences, University of Western Australia, Perth, WA, Australia, Stem Cell Transplant and have published the Article: Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism, in the Journal: (JOURNAL)

SUMMARY

    HSCT is an option and even standard of care for specific metabolic diseases, where other available therapies are less effective and where the benefit of HSCT outweighs the risk of a transplant. This chapter will serially discuss the use of HSCT in certain lysosomal storage and peroxisomal diseases where HSCT is standard . . .

     

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