Novel Molecular and Genetic Therapies for Inherited Arrhythmia Syndrome and Arrhythmogenic Cardiomyopathies-Novel Molecular & Genetic Therapies for Inherited Arrhythmia Syndrome & Arrhythmogenic Cardiomyopathies RT Powered by Solid Biosciences

Novel Molecular & Genetic Therapies for Inherited Arrhythmia Syndrome & Arrhythmogenic Cardiomyopathies RT Powered by Solid Biosciences

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Video Summary

This expert panel discussion, hosted by Solid Biosciences at HRX 2025, explores the integration of emerging molecular genetic therapies for inherited heart diseases into clinical practice. The conversation highlights balancing risk-benefit profiles for providers and patients, emphasizing personalized, shared decision-making given variable disease progression and phenotypic presentation. Key challenges include addressing the cognitive and psychological burden faced by patients and families, especially since these conditions often lack daily symptoms but carry significant uncertainty and fear of sudden cardiac events.<br /><br />Panelists stress the importance of multidisciplinary teams—including genetic counselors and psychologists—and developing disease-specific outcome metrics encompassing physical, emotional, and behavioral aspects. They emphasize ongoing innovation not only in therapy development (e.g., improved gene delivery vectors) but also in clinical implementation, including infrastructure for monitoring and patient support. The unpredictability of future treatment improvements raises complex timing decisions for patients considering irreversible, one-time gene therapies.<br /><br />Real-world considerations, such as insurance, accessibility, and family dynamics (e.g., the "sleepover conundrum"), are discussed. Overall, the experts advocate for thoughtful, patient-centered approaches that recognize the nuanced psychological and clinical impacts of inherited cardiac conditions, aiming for therapies that safely restore function and improve quality of life while navigating uncertainties inherent in pioneering treatments.

Keywords

molecular genetic therapies

inherited heart diseases

personalized medicine

shared decision-making

multidisciplinary teams

gene therapy implementation

psychological impact

patient-centered care