Preferred Label : Eplontersen Sodium;
NCIt definition : The sodium salt form of eplontersen , an antisense oligonucleotide (ASO) targeting
transthyretin (TTR) that is conjugated to three N-acetyl galactosamine (GalNAc) residues,
which can be used in the treatment of polyneuropathy of hereditary TTR-mediated amyloidosis
(hATTR amyloidosis). Upon subcutaneous administration, eplontersen targets and binds
to messenger RNA (mRNA) for both variant and wild-type forms of TTR inside liver cells,
thereby inhibiting translation of both mutant and wild-type TTR. Inhibition of TTR
protein synthesis lowers TTR blood levels and decreases the amount of and/or prevents
TTR amyloid deposits, which accumulate in and cause damage to various body organs
and tissues. ATTR is caused by mutations in the TTR gene, which lead to TTR protein
misfolding. Misfolded wild-type and mutant forms of TTR protein accumulate in tissues
as amyloid deposits in most ATTR patients. The GalNAc residues enable delivery of
the ASO to hepatocytes.;
UNII : WSP2DHR2BD;
CAS number : 2131025-75-5;
Drug name : Wainua;
NCI Metathesaurus CUI : CL1412428;
Origin ID : C174936;
UMLS CUI : C5421032;
Currated CISMeF NLP mapping
- Semantic type(s)
- concept_is_in_subset
