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Differential Transthyretin Stabilization in Patients With Wild Type and Variant Transthyretin Amyloidosis - PubMed

5 hours ago
  • #TTR amyloidosis
  • #pharmacodynamics
  • #cardiomyopathy treatment
  • Acoramidis showed a greater increase in serum TTR (sTTR) compared to placebo plus tafamidis in the ATTRibute-CM trial after 30 months.
  • Acoramidis achieved higher TTR stabilization rates (90.2% vs 60.6% by Western blot) than tafamidis, though sample sizes were small.
  • In vitro analysis confirmed acoramidis as a more effective stabilizer across 51 samples representing 17 unique TTR variants.
  • The study concludes acoramidis is a near-complete stabilizer for both wild type and variant TTR, but direct comparative clinical trial data are lacking.