Metabolic Disorders and Stem Cell Transplantation

Metabolic disorders are inherited genetic defects that interfere with the body’s metabolism, or the process by which the body gets energy from food. Symptoms vary from syndrome to syndrome, but often include developmental delays, vision and hearing problems, loss of intellectual function, muscle weakness, seizures, abnormal movements, stunted growth, pain and shortened life span. A successful bone marrow or stem cell transplant may help slow or halt the progression of certain syndromes, but early diagnosis is critical to prevent irreversible disease progression.

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