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

Adams, David; Polydefkis, Michael; Gonzalez-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt; Schmidt, Hartmut; Berk, John; Losada Lopez, Ines; Dispenzieri, Angela; Quan, Dianna; Conceicao, Isabel; Slama, Michel; Gillmore, Julian; Kyriakides, Theodoros; Ajroud-Driss, Senda; Waddington-Cruz, Marcia; Mezei, Michelle; Plante-Bordeneuve, Violaine; Attarian, Shahram; Mauricio, Elizabeth; Brannagan, Thomas; Ueda, Mitsuharu; Aldinc, Emre; Wang, Jing; White, Matthew; Vest, John; Berber, Erhan; Sweetser, Marianne; Coelho, Teresa

doi:10.1016/s1474-4422(20)30368-9

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

Adams D., Polydefkis M., Gonzalez-Duarte A., Wixner J., Kristen A., Schmidt H. H., ...Daha Fazla

LANCET NEUROLOGY, cilt.20, sa.1, ss.49-59, 2021 (SCI-Expanded, Scopus)

Yayın Türü: Makale / Tam Makale
Cilt numarası: 20 Sayı: 1
Basım Tarihi: 2021
Doi Numarası: 10.1016/s1474-4422(20)30368-9
Dergi Adı: LANCET NEUROLOGY
Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, CINAHL, EMBASE, MEDLINE, Psycinfo
Sayfa Sayıları: ss.49-59
İstanbul Üniversitesi Adresli: Hayır

Özet

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.