Inotersen

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Inotersen (pronunciation: /ɪˈnoʊtərsɛn/) is a medication used for the treatment of hereditary transthyretin amyloidosis (hATTR). It is an antisense oligonucleotide that works by decreasing the amount of transthyretin produced by the body, thereby reducing the formation and deposition of amyloid in tissues.

Etymology

The name "Inotersen" is derived from the Greek words 'ino' meaning 'sinew' or 'nerve', and 'tersen' meaning 'cutting back', reflecting the drug's mechanism of action in reducing the production of transthyretin.

Pharmacology

Inotersen is an antisense oligonucleotide that binds to the messenger RNA (mRNA) of the transthyretin gene. This binding leads to the degradation of the mRNA, thereby reducing the production of transthyretin protein. The reduction in transthyretin protein levels can help to slow the progression of hATTR.

Usage

Inotersen is used for the treatment of stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis. It is administered by subcutaneous injection.

Side Effects

Common side effects of Inotersen include injection site reactions, nausea, headache, fatigue, and thrombocytopenia. Serious side effects may include kidney disease and stroke.

See Also

External links

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