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Transthyretin Amyloid Cardiomyopathy Treatment: An Updated Review
Dinusha Wanniarachchige1, Shazli Khan2, Stephen Pan1,3
1School of Medicine, New York Medical College, Valhalla, NY 10595, USA.
Journal of Clinical Medicine
|September 13, 2025
Summary
Novel therapies are transforming transthyretin amyloid cardiomyopathy (ATTR-CM) treatment. Stabilizers and silencers improve patient outcomes, while gene-editing and antibody therapies show future promise for this heart failure cause.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive restrictive cardiomyopathy.
- ATTR-CM results from misfolded transthyretin protein deposition.
- Historically, liver transplantation was the primary treatment option.
Purpose of the Study:
- To review current and emerging therapeutic strategies for ATTR-CM.
- To investigate literature and clinical trials for ATTR-CM treatments.
- To explore personalized medicine approaches for ATTR-CM.
Main Methods:
- Literature review of existing studies.
- Analysis of ongoing clinical trials.
- Evaluation of novel therapeutic agents.
Main Results:
- Transthyretin (TTR) stabilizers (tafamidis, acoramidis) and TTR silencers (vutisiran) improve survival, function, and quality of life.
- Gene-editing and monoclonal antibody therapies show promise in early studies for amyloid reduction.
- Current standard therapies include stabilizers and silencers.
Conclusions:
- Novel therapies have made ATTR-CM a manageable disease.
- TTR stabilizers and silencers are effective standard treatments.
- Gene-editing and monoclonal antibodies represent promising future therapeutic avenues for ATTR-CM.
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