Summary Summary Short summary of a recent publication, written by scientific experts.

Long-term outcomes of patisiran for transthyretin-mediated amyloidosis with polyneuropathy

Takeaway

  • Patisiran (PN) appears to maintain efficacy with an acceptable safety profile in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRvPN).

Why this matters

  • ATTRvPN is a rare, progressive disease caused by transthyretin (TTR) gene mutations, whereby misfolded TTR accumulates as amyloid deposits in the organs and tissues; and patisiran (RNA interference agent) inhibits hepatic TTR synthesis, reducing serum levels.

    • The maintained efficacy and safety profile of PN provides a viable treatment option for patients with ATTRvPN, potentially improving quality of life; however, continued long-term surveillance is necessary for overall safety evaluation.

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Genetics Patisiran Amyloidosis Hereditary transthyretin-mediated amyloidosis with polyneuropathy Neuropathy Impairment Score Open-label extension trial