Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line

被引：44

作者：

Dowling, James J. ^{[1
,2
]}

机构：

[1] Hosp Sick Children, Div Neurol, 555 Univ Ave, Toronto, ON M5G 1X8, Canada

[2] Hosp Sick Children, Program Genet & Genome Biol, 555 Univ Ave, Toronto, ON M5G 1X8, Canada

来源：

NATURE REVIEWS NEUROLOGY | 2016年 / 12卷 / 12期

关键词：

D O I：

10.1038/nrneurol.2016.180

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

New therapies are much needed for Duchenne muscular dystrophy. Recent data from a phase II clinical trial has led to accelerated FDA approval of the exon-skipping drug eteplirsen. This approval is provisional, pending results of an ongoing phase III clinical trial, and came after much debate.

引用

页码：675 / 676

页数：2

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