Lysosomal glycogen storage disease without acid maltase deficiency

Abstract
Two brothers with lysosomal glycogen storage disease without acid maltase deficiency in skeletal muscle were studied. Although no specific biochemical defect was identified, a characteristic clinical picture emerged from evaluation of these siblings and 2 other previously reported patients. The syndrome is manifested by proximal muscle weakness, hypertrophic cardiomyopathy, probable intellectual impairment and possible liver involvement.