NCIt definition : An antisense oligonucleotide (ASO) targeting transthyretin (TTR), which has potential
use in the treatment of TTR amyloidosis (ATTR). Upon subcutaneous administration,
inotersen 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.;
UNII : 0IEO0F56LV;
CAS number : 1492984-65-2;
Molecule name : ISIS-TTR Rx; ISIS-TTRRx; ISIS 420915;
https://www.has-sante.fr/jcms/p_3106176/fr/tegsedi 2019 false false false France inotersen Inotersen evaluation of the transparency committee Inotersen oligonucleotides