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Hepatic Glycogen Storage Disease<subtitle>CLINICAL AND LABORATORY FINDINGS IN 23 CASES</subtitle>

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1971

Year

Abstract

Clinical and laboratory findings and the results of enzyme studies made on tissue samples from 23 children with hepatic glycogen storage disease are described. Two-thirds of the patients had glycogenosis associated with deficiency of glucose-6-phosphatase, amylo 1,6-glucosidase, or hepatic phosphorylase. No enzyme defect could be found to account for excessive glycogen deposition in the others, who would have been incorrectly diagnosed as suffering from one of the recognized enzyme deficiencies, if samples of affected tissue had not been examined for specific enzyme activities. The treatment of glycogen storage disease has been limited to regulating the blood sugar level and preventing hypoglycaemia. In most patients this was achieved by simple dietary management. Two children who did not respond to dietary control have been treated with diazoxide.