医学
多发性神经病
内科学
基因
疾病
免疫学
基因组编辑
生物信息学
突变
抗体
遗传学
作者
Julian D. Gillmore,Ed Gane,Jörg Täubel,Björn Pilebro,Andoni Echaniz‐Laguna,Justin Kao,William J. Litchy,Safi Shahda,Alexandra Haagensen,Liron Walsh,Derek K. Smith,Jessica Kachadourian,John H. Ward,David Lebwohl,Peijuan Zhu,Yuanxin Xu,Adia Leung,Alison Sonderfan,David E. Gutstein,Garen Manvelian
标识
DOI：10.1056/nejmoa2510209
摘要
A single administration of nex-z in patients with ATTRv-PN was associated with rapid, deep, and durable reductions in serum TTR levels. The results support further investigation of nex-z to treat ATTRv-PN. (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).
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