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...

Volledige beschrijving

Bibliografische gegevens

Hoofdauteurs:	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
Formaat:	Artigo
Taal:	Inglês
Gepubliceerd in:	Taylor & Francis Group 2025-12-01
Reeks:	Annals of Medicine
Onderwerpen:	ATTRv amyloidosis patisiran polyneuropathy RNA interference treatment
Online toegang:	https://www.tandfonline.com/doi/10.1080/07853890.2025.2537347
Tags:	Voeg label toe Geen labels, Wees de eerste die dit record labelt!