Definitive therapy for PGK deficiency necessarily involves production of the PGK enzyme. The hemolytic anemia that is part of this disease could be cured by allogeneic stem cell transplant, as replacing the hematopoietic system would ensure production of the enzyme in the red blood cells. However, the neurologic complications of the disease would require that the enzyme can find its way into the neurons themselves. Because of the rarity of PGK deficiency, there has been as yet no controlled study to define the most efficacious approach. In fact, there is no mention of any therapy in the literature. There is currently no enzyme replacement therapy and no major research effort underway to do so. To our knowledge, stem cell transplant in this disease has not been performed except for in one 7 month old child with PGK-Amiens in Australia, who is currently being prepared for SCT. The stem cell source will be determined by the donor availability. The donor registry allows the donor the option of donating either peripheral blood or bone marrow.