An RNA interference compound that halts or reverses poly neuropathy due to  hereditary transthyretin amyloidosis in adults.

Tradename Amvuttra.

It is a modified double-stranded small interfering RNA that targets variant and wild type TTR messenger RNA in the liver.

It reduces TTR serum levels in tissue deposits, which are thought to cause polyneuropathy.

It is injected subcutaneously once every three months.

Treated patients showed significant improvement on a neuropathy impairment scale, with improve quality of life, nutritional status and 10 minute walking test results compared with placebo.

Common adverse events were arthralgia, dyspnea, and decreases vitamin A levels. Patients who take vitrisiran  should also take vitamin A supplements.


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