Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Date
2021
Author
Coelho, Teresa
Adams, David
Polydefkis, Michael
Gonzalez-Duarte, Alejandra
Wixner, Jonas
Kristen, Arnt
White, Matthew T.
Vest, John
Berber, Erhan
Sweetser, Marianne T.
Schmidt, Hartmut H.
Berk, John L.
Losada Lopez, Ines Asuncion
Dispenzieri, Angela
Quan, Dianna
Conceicao, Isabel M.
Slama, Michel S.
Gillmore, Julian D.
Kyriakides, Theodoros
Ajroud-Driss, Senda
Waddington-Cruz, Marcia
Mezei, Michelle M.
Plante-Bordeneuve, Violaine
Attarian, Shahram
Mauricio, Elizabeth
Brannagan, Thomas H.
Ueda, Mitsuharu
Aldinc, Emre
Wang, Jing Jing
Metadata
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Abstract
Background Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy.
URI
http://hdl.handle.net/20.500.12627/179130
https://doi.org/10.1016/s1474-4422(20)30368-9
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