Spinal muscular atrophy - challenges in the therapeutic era

被引：1
|
作者：
Faravelli, Irene [1 ]
Corti, Stefania [1 ,2 ]
机构：
[1] Univ Milan, Dino Ferrari Ctr, Neurosci Sect, Dept Pathophysiol & Transplantat DEPT, Milan, Italy
[2] Fdn IRCCS Ca Granda Osped Maggiore Policlin, Neurol Unit, Milan, Italy
来源：
NATURE REVIEWS NEUROLOGY | 2020年 / 16卷 / 12期
关键词：
NUSINERSEN;
D O I：
10.1038/s41582-020-00411-6
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
With the advent of disease-modifying therapies for spinal muscular atrophy, prenatal and extra-neural alterations associated with the condition have garnered increasing attention as potential determinants of the therapeutic window and efficacy of novel drugs. Two recent studies highlight the impact of spinal muscular atrophy on prenatal bone and organ development.
引用
收藏
页码：655 / 656
页数：2
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