About Genetic Disorders
(including inborn errors of metabolism, Fanconi’s anemia)

For children with “inborn errors of metabolism” — a variety of genetic diseases characterized by the deficiency of enzymes, resulting in the accumulation of toxic chemicals in the cells — bone marrow transplantation is the only treatment. This is also true for Fanconi’s anemia, a rare genetic disorder characterized by deficiency of all bone marrow elements including red blood cells, white blood cells and platelets.

Treatment for Genetic Disorders

At the Bone Marrow Transplant Center at Rush University Medical Center in Chicago, Illinois, children with inborn errors of metabolism or Fanconi’s anemia who meet specific criteria may be eligible for allogeneic stem cell transplant using a matched sibling, unrelated donor marrow or cord blood.