Lysosomal Diseases and Fibroblast Cultures: Biochemical and Electron Microscopic Observations

Most lysosomal diseases are fatal, often after a protracted downhill course that is painfully apparent to patient, parents and physician. This interim report relates our attempts to treat such patients; and to find ultrastructural and biochemical markers in fibroblast cultures for the study of pathophysiology and treatment of lysosomal disease.