Patisiran

Last updated on: 03.08.2025

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LiteratureThis section has been translated automatically.

Adams D, Gonzalez-Duarte A, O'Riordan WD et al (2018) Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med;379(1):11-21 doi/full/10.1056/NEJMoa1716153.

Adams D, Wixner J, Polydefkis M et al (2025). Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy A Randomized Clinical Trial With Open-Label Extension. JAMA Neurol. 82(3):228-236. https://jamanetwork.com/journals/jamaneurology/fullarticle/2828606.

Schmidt HH, Wixner J, Planté-Bordeneuve V et al (2022). Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation. Am J Transplant. 22(6):1646-1657. doi: 10.1111/ajt.17009.

Patisiran ^Onpattro® (Alnylam)

Last updated on: 03.08.2025