Disease-modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications.

ABSTRACT: Transthyretin amyloidosis (ATTR) is a rare disease that results in amyloid fibril misfolding and deposition in multiple organs, including the heart, leading to the development of ATTR cardiomyopathy (ATTR-CM), which is associated with poor outcomes. In the last decade, several disease-modifying medications are in advanced stages of clinical development or have been approved to treat ATTR-CM. The purpose of this review is to critically evaluate clinical trial data investigating the use of approved and investigational medications for the treatment of ATTR-CM. We performed a comprehensive literature search via PubMed and EMBASE to identify randomized controlled trials evaluating medications for the treatment of ATTR-CM published through August 2024. This narrative review describes the pathoph

SUBMITTER: Hellenbart EL 

PROVIDER: S-EPMC11823349 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature