The development of pharmacologic agents for the treatment of diseases is still challenging, especially in rare inherited arrhythmia syndromes like long and short QT syndrome, Brugada syndrome and catecholaminergic polymorphic ventricular tachycardia. The underling pathophysiologic mechanism of these inherited diseases is in most cases either a gain- or a loss-of-function due to mutations in ion channel genes. Although the biophysical properties of mutant channel subunits are well studied, little is known about the targeting effect of specific pharmacologic agents. In this review, we focus on the therapeutic (in vivo) and the experimental (in vitro) approaches in the most common inherited forms.