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

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ID: 334095
2026
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Abstract
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.
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Authors Saranda Nimani, Katja E. Odening
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Journal european heart journal
Year 2026
DOI
10.1093/eurheartj/ehag795
URL
https://doi.org/10.1093/eurheartj/ehag795
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