Advances in the Treatment of Transthyretin Cardiac Amyloidosis: Current and Emerging Therapies

Transthyretin cardiac amyloidosis (ATTR-CA) has been recognized as an underdiagnosed and undertreated cause of heart failure with often unrecognized multiorgan involvement. Guideline development and the establishment of nonbiopsy criteria for diagnosis of ATTR-CA has led to an increased rate of diagnosis and hence patients referred for therapies. ATTR is a protein misfolding disorder where the TTR tetramer disassociates into monomers which form insoluble amyloid depositions in organs, including the heart. ATTR-CA can be due to autosomal dominant transmitted gene mutation or due to misfolding of wild type TTR. Prior to 2019, there were no FDA-approved pharmacological treatments for ATTR-CA. Understanding of ATTR-CA pathogenesis has enabled development of targeted strategies with novel disease-modifying therapies. Current and emerging therapies for ATTR-CA include: 1) TTR gene silencing (siRNA, ASO, CRISPR/Cas9) 2) TTR tetramer stabilization and 3) TTR amyloid fibril degradation. This review focuses on the pathophysiology of ATTR-CA, diagnostic criteria, and addresses current and emerging treatments for the diverse disorder.

留言 (0)

沒有登入
gif