Research advances since the turn of the century have identified new pediatric disorders, uncovered the genetic and molecular basis for many more disorders, and have led to progression from gene discovery to effective small molecule therapies that rectify molecular dysfunction.1 The emergence of biologic therapies as well as new drugs has improved health outcomes for children with autoimmune diseases, cancer, and other disorders.2 Pluripotent stem cell–based regeneration of tissues, such as intestinal organs,3 is a laboratory advancement that holds promise for pediatric application.