CLINICAL, BIOCHEMICAL AND ULTRASTRUCTURAL STUDIES OF AN ATYPICAL FORM OF MUCOPOLYSACCHARIDOSIS

Abstract
Summary: Two children, brother and sister, presenting a clinically peculiar form of mucopolysaccharidosis are reported. There exists a discrepancy between clinical and radiological data and biochemical findings. Enzyme study of the liver discloses a striking hyperactivity of the acid /J‐galactosidase. Hepatic ultrastructure differs from that of other previous descriptions of mucopolysaccharidoses, demonstrating a complex storage of lipids and mucopolysaccharides within swollen lysosomes.