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.
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Advancements in ATTR Amyloidosis
- ATTR amyloidosis has seen significant advancements in diagnosis and treatment.
- It's now recognized as not rare, especially in older heart failure patients, and multiple treatments exist.
Managing ATTR Amyloidosis
- Manage ATTR amyloidosis patients with diuretics, MRAs, and SGLT2 inhibitors.
- Consider reducing beta-blockers unless needed for arrhythmia rate control.
Amyloid-Specific Therapies
- Consider Tafamidis or Acaramitis for amyloid-specific therapy in ATTR-CM.
- Earlier diagnosis leads to better treatment outcomes with these therapies.