Liver transplantation for type I glycogen storage disease.

STUDIES of haptoglobin, 1-3 group-specific component,3,4 and numerous other products of hepatic synthesis59 have shown that liver homografts permanently retain their original metabolic specificity after transplantation. Consequently, liver transplantation has been regarded for some years4 as a potentially decisive way to treat those inborn errors of metabolism which result partly or completely from defects in hepatic function. This objective has been realised in several different metabolic disorders.8,9 We report here the first use of liver transplantation for the treatment of type I glycogen storage disease, a disorder caused by glucose-6-phosphatase deficiency .