Why dystrophin quantification is key in the eteplirsen saga

被引：19

作者：

Aartsma-Rus, Annemieke ^{[1
]}

Arechavala-Gomeza, Virginia ^{[2
]}

机构：

[1] Leiden Univ, Med Ctr, Dept Human Genet, Leiden, Netherlands

[2] Biocruces Hlth Res Inst, Neuromuscular Disorders Grp, Baracaldo, Spain

来源：

NATURE REVIEWS NEUROLOGY | 2018年 / 14卷 / 08期

关键词：

D O I：

10.1038/s41582-018-0033-8

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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.

引用

页码：454 / 456

页数：4

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