Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study

Background This study assessed the effectiveness and safety of patisiran in patients with V122I/T60A variant transthyretin (ATTRv) amyloidosis with polyneuropathy. These variants have been under-represented in previous trials of gene-silencing agents.Methods This was a multicenter, phase IV study co...

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Autors principals:	Yessar Hussain, Saurabh Malhotra, Brett W. Sperry, Francy Shu, Karthikeyan Ananthasubramaniam, Urvi Desai, Ugochukwu Egolum, Joel Fernandez, Miriam Freimer, Elizabeth Mauricio, Dianna Quan, Roy Small, Kelley Capocelli, Steven Roblin, Shaun Bender, Patrick Y. Jay, Elena Yureneva, Ronald Zolty
Format:	Artigo
Idioma:	Inglês
Publicat:	Taylor & Francis Group 2025-12-01
Col·lecció:	Annals of Medicine
Matèries:	ATTRv amyloidosis patisiran polyneuropathy RNA interference treatment
Accés en línia:	https://www.tandfonline.com/doi/10.1080/07853890.2025.2537347
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