Why the study?
Diagnosing ATTR remains challenging, especially in asymptomatic variant carriers, and management involves complex therapeutic dilemmas with a limited range of conventional therapies.
Emerging therapies such as RNA-based treatments, CRISPR-Cas9 gene editing, and monoclonal antibodies offer promising new approaches for managing ATTR cardiomyopathy.
These therapies remain investigational in ATTR cardiomyopathy; leaves open the need for randomized trials before clinical adoption.
Transthyretin amyloidosis (ATTR) manifests as wild-type (ATTRwt) and hereditary/mutant (ATTRv) forms and can lead to heart failure due to cardiac amyloidopathy.Diagnosing ATTR, particularly in asymptomatic carriers of pathogenic variants, remains challenging despite the advances.Complex and multi-aspect management involves a limited range of well-examined conventional therapies to address the heart failure and frequently coexisting arrhythmias and valvular issues.Disease-modifying treatment, RNA-based treatments, CRISPR-Cas9 gene editing and monoclonal antibodies targeting amyloid deposits are recent and promising innovations.This review explores the diagnostic intricacies, therapeutic dilemmas and emerging solutions in ATTR cardiomyopathy.The significance of early detection and precise, targeted approaches to enhance patient outcomes is underscored.
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Kulak et al. (2024) studied this question.
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