Migraine is a paroxysmal neurological disorder affecting up to 12% of males and 24% of females in the general population, demonstrated to have a strong, but complex, genetic component. Genetic investigation of migraine bears great promise in providing new targets for drug development and optimization of individual specific therapy. Better, preferably prophylactic, treatment of migraine patients is desired because the presently used drugs are not effective in all patients, allow recurrence of the headache in a high percentage of patients and sometimes have severe adverse side effects. With the recent identification of the brain-specific P/Q-type calcium channel gene CACNA1A in the pathogenesis of migraine, the first step has been taken to identify primary biochemical pathways leading to migraine. Here, we summarize the current knowledge about the genetics of migraine and focus on the implication for treatment approaches.