Targeting trafficking defects in long QT syndrome type 2: a phase 2 clinical study with patient-specific cellular validation. (PubMed, Eur Heart J) - Sep 23, 2026 - "Short-term LUM therapy is safe and yields a significant QTc shortening in LQT2 patients with a trafficking defect. Concordance between cellular and clinical findings reinforces the value of a mechanism-based, variant-informed approach and supports the feasibility of trafficking-correction strategies within a precision-medicine framework for inherited arrhythmia syndromes."
Journal • P2 data • Cardiovascular • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • KCNH2
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Annarita Di Mise; Carla Spazzolini; Chiara Alberio; Federica Dagradi; Fulvio L F Giovenzana; Giulia Musu; Lia Crotti; Luca Sala; Manuela Mura; Massimiliano Gnecchi; Matteo Pedrazzini; Miriam G Lorusso; Paolo Cerea; Peter J Schwartz; Prabin Upadhyaya
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