Transplantation as disease modifying therapy in adults with inherited metabolic disorders
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DOI: 10.1007/s10545-018-0141-z
Abstract: Transplantation is an established disease modifying therapy in selected children with certain inherited metabolic diseases (IMDs). Transplantation of hematopoietic stem cells or solid organs can be used to partially correct the underlying metabolic defect, address… read more here.
Keywords: disease modifying; disease; inherited metabolic; transplantation ... See more keywords