Enriched hematopoetic stem cell for inherited metabolic disorders
This research study attempts to establish chimerism in order to prevent graft-versus-host disease in patients with inherited metabolic disorders. Hematopoeitic stem cells are transplanted into the patients establishing chimerism. The 7-year probability of overall survival was 90% (95% CI, 86%-93%).
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Enriched hematopoetic stem cell for inherited metabolic disorders