Joanne Kurtzberg, MD – Inborn Errors of Metabolism Disorders

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The objective of this study is to make T-cell depleted stem cells from a family member who
is a half match (haplo-identical) available on an expanded access basis to patients
receiving one or two unrelated cord blood transplants who are at a higher risk of not
engrafting in a safe amount of time. The purpose of the related stem cells is the give the
bone marrow a “jump start” towards recovery. Ultimately, the cord blood cells will grow and
permanently rescue the bone marrow.