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Cardionerds: A Cardiology Podcast
407. Journal Club: The Nex-Z Trial – A CRISPR-Cas9 Based Treatment for ATTR Cardiac Amyloidosis with Dr. Ronald Witteles
Jan 16, 2025
Dr. Ronald Witteles, an Advanced Heart Failure and Transplant Cardiologist at Stanford, dives into the promising Nex-Z trial investigating a CRISPR-Cas9 treatment for ATTR cardiac amyloidosis. Listeners will learn how this innovative gene-editing technology shows potential for effective therapies targeting protein misfolding. Witteles discusses the trial's safety results, significant TTR level reductions, and anticipates future applications, shedding light on the evolution of heart failure treatments and the hope for one-time therapies.
19:23
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Quick takeaways
- The Nex-Z trial showcased a promising CRISPR-Cas9 treatment that significantly reduced serum TTR protein levels in ATTR cardiomyopathy patients.
- Emerging therapies like tafamidis and acaramidis are reshaping management strategies for ATTR, highlighting significant progress in previously challenging conditions.
Deep dives
Advancements in ATTR Treatment Options
Recent advancements in the treatment of ATTR cardiomyopathy have significantly changed management strategies. The approval of tafamidis in 2019 marked a turning point, offering patients a substantial treatment effect, especially when diagnosed early. Following this, acaramidis has emerged as a promising agent that stabilizes transthyretin proteins, reducing cardiovascular hospitalizations by approximately 50%. These therapies, alongside newer developments, exemplify the progress made in addressing a previously challenging and often overlooked condition.
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