Basic Information
ID DDInter926
Drug Type biotech
Protein Chemical Formula C230H318N69O121P19S19
Protein Average Weight 7183.077
CAS Number 1492984-65-2
Description Inotersen is a transthyretin-directed antisense oligonucleotide for the treatment of the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults. It was FDA approved in October 2018.[L11761] Inotersen has been shown to improve the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis [A39493]. Hereditary transthyretin amyloidosis is caused by single-nucleotide variants in the gene encoding transthyretin (TTR), which lead to transthyretin misfolding and the deposition of amyloid substance systemically. Progressive amyloid accumulation may lead to multiorgan dysfunction and death [A39493].
ATC Classification N07XX15
Sequences None
Useful Links DrugBank Wikipedia
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