Scholarlink

In the past decade, treatment of transthyretin cardiomyopathy (ATTR-CM) has been revolutionized mainly due to the deepened understanding of the cellular biology of the disease. Intervening in TTR synthesis, by gene editing or RNA silencing, TTR tetramer stabilization and TTR amyloid removal from tissues could all serve as therapeutic targets. There are now three FDA approved disease modifying therapies for ATTR-CM. These therapies targeting different mechanisms in the pathophysiology of ATTR-CM provide new alternatives for treating the disease. This chapter will discuss available and investigational therapies, their mechanism of action, indications, evidence for their efficacy, and safety profile. Finally, this chapter will summarize all major completed and ongoing trials for the treatment of ATTR-CM.

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

New Frontiers and Evolving Therapies in ATTR Cardiac Amyloidosis

  • 章节

  • 发表日期：

    2025年

查看全文
  • Abdirahman Wardhere,
  • Dimitrios Bampatsias,
  • Mathew S. Maurer

摘要

In the past decade, treatment of transthyretin cardiomyopathy (ATTR-CM) has been revolutionized mainly due to the deepened understanding of the cellular biology of the disease. Intervening in TTR synthesis, by gene editing or RNA silencing, TTR tetramer stabilization and TTR amyloid removal from tissues could all serve as therapeutic targets. There are now three FDA approved disease modifying therapies for ATTR-CM. These therapies targeting different mechanisms in the pathophysiology of ATTR-CM provide new alternatives for treating the disease. This chapter will discuss available and investigational therapies, their mechanism of action, indications, evidence for their efficacy, and safety profile. Finally, this chapter will summarize all major completed and ongoing trials for the treatment of ATTR-CM.