Eplontersen - CAS 1637600-16-8

Eplontersen, a triantennary N-acetyl galactosamine (GalNAc3-7a) -conjugated antisense oligonucleotide, targets transthyretin (TTR) mRNA to suppress the synthesis of both variant and wild-type TTR proteins. The accumulation of misfolded TTR proteins contributes to the formation of amyloid fibrils in the heart and peripheral nerves, resulting in amyloid TTR (ATTR) amyloidosis [1] [2] [3].

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