期刊
NATURE REVIEWS NEUROLOGY
卷 14, 期 8, 页码 454-456出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/s41582-018-0033-8
关键词
-
Eteplirsen, a compound designed to restore dystrophin in patients with Duchenne muscular dystrophy, controversially received approval by the FDA in 2016. Owing to limited clinical data, the approval was based on eteplirsen's effect on dystrophin expression. Now, the dystrophin quantification results have been published, and although low levels of dystrophin expression are shown, the quantification remains debatable.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据