DMD and Duchenne muscular dystrophy: Very little is known about the functional role of dystrophin in the brain, however, animal models of DMD exhibit a wide range of Purkinje cell deficits in excitability and plasticity, consistent with the high expression of dystrophin in this cell population.4 Dystrophin is highly expressed in cerebellar Purkinje neurons in addition to being expressed in other areas of the central and peripheral nervous systems3 (Supplementary Figure 1).