[Transthyretin amyloid cardiomyopathy]

Area of Science:

• Cardiology
• Genetics
• Pharmacology

Background:

• Transthyretin amyloidosis (ATTR-CM) is a progressive infiltrative cardiomyopathy.
• Amyloid fibril deposition in ATTR-CM leads to heart failure, arrhythmias, and mortality.
• ATTR-CM prevalence is increasing globally.

Purpose of the Study:

• To review the current landscape of ATTR-CM.
• To discuss the impact of newly approved and emerging therapies.
• To highlight the potential of future treatments to alter disease trajectory.

Main Methods:

• Literature review of ATTR-CM.
• Analysis of current and pipeline therapeutic agents.
• Discussion of clinical trial data and outcomes.

Main Results:

• The first disease-modifying drugs for ATTR-CM are now available in Denmark.
• Several additional treatments are awaiting clinical implementation.
• Novel therapies in clinical trials aim to halt or reverse ATTR-CM progression.

Conclusions:

• Emerging therapies promise to significantly improve morbidity and mortality associated with ATTR-CM.
• The advent of disease-modifying and potentially curative treatments marks a new era in ATTR-CM management.
• Continued research and clinical trials are crucial for advancing ATTR-CM care.