Migraine is a prevalent, debilitating and costly disorder with an ongoing unmet medical need. Human genetic studies have provided considerable insights into the molecular underpinnings of this complex brain disorder. Classical linkage studies have revealed the causes of familial hemiplegic migraine, while more recently genome-wide association studies have identified several susceptibility loci for typical migraine. New ways of accessing neurons and other cells directly from patients with migraine through the use of induced pluripotent stem cells offer exciting opportunities to understand the molecular pathogenesis. In conjunction with next generation omics, there are unprecedented opportunities to reveal key molecular players in the disease process and discover new drug targets.