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Article

Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis

23 Jul 2022

Abstract excerpt

Aims Transthyretin‐mediated amyloidosis is a progressive and fatal disease caused by the build‐up of misfolded transthyretin (TTR) protein. Eplontersen is a triantennary N‐acetyl galactosamine (GalNAc3)‐conjugated antisense oligonucleotide targeting TTR messenger ribonucleic acid (mRNA) to inhibit production of both variant and wild‐type TTR. We aimed to develop a population pharmacokinetic/pharmacodynamic...

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PMID: 35869634
DOI: 10.1111/bcp.15468

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