Stem Cell Transplantation in Inherited Metabolic Disorders

doi:10.1182/asheducation-2011.1.285

Article

Stem Cell Transplantation in Inherited Metabolic Disorders

7 Dec 2011

Abstract excerpt

Allogeneic HSCT is performed for a small number of inborn errors of metabolism (IEM). Over the last years, transplantation outcomes have improved in this group of patients as the factors that predicted for poor transplantation outcomes were understood and addressed. The role of transplantation and its potential benefit for an individual patient with a certain IEM is therefore now much better defined. In parallel...

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