7 #AHA24 ScientificSessions.org for the medical community — from physicians and physician-scientists to pharmaceutical companies — to look toward diseases with effective therapies or interventions for patients and have a subconscious desire to focus efforts in those areas. In rare cardiomyopathies, the picture is further complicated because fewer patients have been identified, resulting in less data to rely on. Creating well-designed interventional trials is another challenge, she said. Hurdles include an evolving knowledge of the natural history of these cardiomyopathies; pace of developing validated fit-forpurpose diagnostic tools specific for these conditions; evolving diagnostic algorithms; lack of disease-centric endpoints for clinical trials; and limited interest in investing to advance the science in rare diseases. Nevertheless, Adigun noted several management strategies to improve outcomes for cardiomyopathies. They include: • Enhanced screening and surveillance in at-risk populations, providing the opportunity to continue educating patients and health care professionals • Disease-modifying therapies or other interventions, when available • Symptom management and prevention of disease-related complications Like Alexander, Adigun said she believes cardiology is seeing great strides in precision medicine and, although this will affect management of diseases generally, the impact will be more evident in rare cardiomyopathies. Advances in genome sequencing, evolution of cardiac imaging modalities and novel work in creating in-vitro and exvivo models of disease can improve understanding of the molecular underpinnings of disease and identify better targets for interventions, she said. “With genomic sequencing, we can now identify more cardiovascular diseases that have a defined inheritance pattern caused by single genetic variant compared to other common cardiovascular diseases characterized by accumulation of small genetic variants with different implications,” Adigun said. “Identifying these variants and their weighted implications for disease manifestations are informing research in genome editing modalities in inherited cardiomyopathies, with encouraging data in early phase studies.” Join us in celebrating the 100th year of the American Heart Association by igniting the future of science! Your donation fuels groundbreaking innovation and discovery, helping us continue our mission to fight heart disease and stroke. Every contribution counts. Let’s make history! Scan the QR code to make a difference. *Donate $100 or more and select a free gift from the on-site Shop Heart store located in the registration area. IGNITE THE FUTURE
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