us with a unique example of disease relevant phenotypes isolated in the dish that are directly translatable to the clinic. Furthermore, this study gives direct evidence that cohorts of patients exhibiting similar clinical phenotypes may exhibit differential response to therapeutics based on that individual’s unique genomic landscape. From a clinical standpoint, iPS cell technology can not only test the efficacy and mechanism of action for widely prescribed medications but also allows developing novel drug candidates to discover new therapies proactively in a species-relevant model system prior to their use in the clinic (Figure 1).