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Amyloid
The Journal of Protein Folding Disorders
Volume 28, 2021 - Issue 2
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Letters to the Editor

Comment to ‘Nelson LT, Paxman RJ, Xu J, et al. Blinded potency comparison of transthyretin kinetic stabilisers by subunit exchange in human plasma’

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Pages 138-139 | Received 27 Sep 2020, Accepted 15 Nov 2020, Published online: 02 Dec 2020

References

  • Nelson LT, Paxman RJ, Xu J, et al. Blinded potency comparison of transthyretin kinetic stabilisers by subunit exchange in human plasma. Amyloid. [cited 2020 Aug 18]; [6 p.]. DOI:10.1080/13506129.2020.180 8783
  • Fox JC, Hellawell JL, Rao S, et al. First‐in‐human study of AG10, a novel, oral, specific, selective, and potent transthyretin stabilizer for the treatment of transthyretin amyloidosis: a phase 1 safety, tolerability, pharmacokinetic, and pharmacodynamic study in healthy adult volunteers. Clin Pharmacol Drug Dev. 2020;9(1):115–129.
  • Sant'Anna R, Gallego P, Robinson LZ, et al. Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity. Nat Commun. 2016;7:10787.
  • Miller M, Pal A, Albusairi W, et al. Enthalpy-driven stabilization of transthyretin by AG10 mimics a naturally occurring genetic variant that protects from transthyretin amyloidosis. J Med Chem. 2018;61(17):7862–7876.
  • Judge DP, Heitner SB, Falk RH, et al. Transthyretin stabilization by AG10 in symptomatic transthyretin amyloid cardiomyopathy. J Am Coll Cardiol. 2019;74(3):285–295.