Long-Term Corticosteroid Therapy in Hurler Syndrome

Introduction The syndrome referred to as the Hurler or Hunter-Hurler syndrome (lipochondrodystrophy or "gargoylism") was first described as a clinical entity in 1917. 1-3 In 1952 Brante 4 identified the "storage substance" in the liver and meninges of three such autopsied individuals to have the characteristics of an acid mucopolysaccharide. He believed this material to be chemically similar or identical to chondroitinsulfuric acid. Dorfman and Lorincz, 5 Brown, 6 and Meyer et al. 7-9 isolated the acid mucopolysaccharide com- ponents from the urine and tissues of individuals with this syndrome. Excessive quantities of chondroitinsulfuric acid-B (B-heparin) and heparitin sulfate were found to be present. In recent years interest has been focused on the relationship of the adrenal corticosteroids to diverse aspects of connective tissue metabolism. Layton 10 found that sulfate fixation in the skin of intact rats was inhibited by cortisone administration. Schiller and Dorfman 11,12 reported that the uptake