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Precision Medicine for Long QT Syndrome Type 2: Lumacaftor Corrects Trafficking Defects and Shortens QT Interval
Cardiologyprecision medicineLong QT syndrome

Precision Medicine for Long QT Syndrome Type 2: Lumacaftor Corrects Trafficking Defects and Shortens QT Interval

By MedXY|Sep 28, 2026

A phase 2 clinical study demonstrates that lumacaftor safely shortens QTc in LQT2 patients with KCNH2 trafficking defects, supported by patient-specific cardiomyocyte models validating the mechanism.

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Precision Medicine for Long QT Syndrome Type 2: Lumacaftor Corrects Trafficking Defects and Shortens QT Interval