An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1.

已完结 10 由 Luke 发布于 2026/6/1 20:42:31

DOI:10.1056/NEJMoa2407326
作者：Nicholas E Johnson,?Li-Jung Tai,?Johanna I Hamel,?John W Day,?Jeffrey M Statland,?Payam Soltanzadeh,?Sankarasubramoney H Subramony,?Charles A Thornton,?W David Arnold,?Matthew Wicklund,?Miriam L Freim
文献类型：期刊论文
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New England Journal of Medicine (NEJM)

Abstract：Background: Myotonic dystrophy type 1 is a rare, dominantly inherited, progressive, disabling, neuromuscular disease that leads to decreased life expectancy and has no approved therapies. Th

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