Polycystic kidney disease (PKD) represents the most common inherited cause of chronic renal failure. PKD is a relatively uncommon cause of chronic renal failure or mortality in childhood and adolescence, but is nevertheless often responsible for symptoms of renal disease. Current research into the pathogenesis of PKD suggests that disturbance of the normal regulation of growth and development of tubular epithelium is intrinsic to cyst formation and growth. Features of cystic epithelium that are analogous to earlier stages of renal development include altered composition of the extracellular matrix, abnormal cell proliferation, and the persistence of a secretory pattern of fluid and electrolyte transport. The potential for early diagnosis and intervention in PKD makes it an area of great interest to the pediatric nephrologist. Animal and in vitro studies have achieved modification of cyst growth by reduction of dietary protein, use of amiloride and its analogs, antagonism of the epidermal growth factor receptor system, anti-inflammatory therapy, and most recently with the use of taxol, an agent that inhibits microtubule assembly. PKD may represent an area in which childhood diagnosis and intervention will have a significant impact on the prevalence of chronic renal failure in adult life.