Abstract 13964: Differential ex vivo Stabilization of Transthyretin by AG10 and Tafamidis in Samples From Patients With Moderate or Severely Destabilizing Mutations
2019
Introduction: Transthyretin (TTR) amyloidosis is a progressive, fatal disease caused by destabilizing TTR mutations and age-related factors. Dissociation of tetrameric TTR initiates protein misfold...
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