From mechanism to medicine: lumacaftor for trafficking-deficient long QT syndrome type 2

Summary of the disease mechanisms underlying LQT2 (upper left box), current conventional therapies (upper mid box), and novel emerging mechanism-based therapeutic approaches, including mexiletine, gene therapy (upper right boxes), and lumacaftor (lower box). ER, endoplasmic reticulum; ICD, implantable cardioverter-defibrillator; late INa, late sodium current; LCSD, left cardiac sympathetic denervation; LQT2, long QT syndrome type 2; mRNA, messenger RNA.