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- Hereditary Transthyretin Amyloidosis: Navigating Heterogeneity Toward Timely Diagnosis and Targeted Therapy
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Medical Article
Introduction (1) Hereditary transthyretin amyloidosis (ATTRv) is a progressive, multisystem protein misfolding disorder caused by pathogenic variants in the transthyretin (TTR) gene. Destabilization of the TTR tetramer promotes monomer dissociation, misfolding, and extracellular amyloid deposition, resulting in cumulative organ dysfunction. Periphe

Hereditary Transthyretin Amyloidosis: Navigating Heterogeneity Toward Timely Diagnosis and Targeted Therapy
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