Reviewing Treatment Options for Cardiac Amyloidosis-2. Current and Emerging Treatment in ATTR-CM

Video Transcription

Video Summary

Dr. Richard Chang's presentation focuses on the treatment landscape for TTR cardiomyopathy, particularly targeted therapies and heart failure GDMT. He discusses the biological mechanism of TTR amyloid, explaining how the liver produces the TTR protein, which can misfold and form amyloid deposits leading to heart complications. Emerging treatments aim to target and stabilize these processes using TTR silencers and stabilizers like tefamidase and acromidase, both FDA approved. CRISPR technology and monoclonal antibodies are also being explored for disease reversal. Clinical trials have shown reductions in mortality and improvement in life quality with these treatments, although challenges remain, such as determining the most effective therapies and combining them optimally. Regarding heart failure GDMT, Dr. Chang highlights benefits from SGLT2 inhibitors and MRAs but notes uncertainty about beta blockers and ACES/inhibitors. Overall, ongoing research aims to enhance early diagnosis and effective treatment.

Keywords

TTR cardiomyopathy

targeted therapies

TTR amyloid

CRISPR technology

clinical trials

heart failure GDMT