A prospective natural history study of type 1 spinal muscular atrophy

被引：8

作者：

Bertini, Enrico ^{[1
]}

Mercuri, Eugenio ^{[2
,3
]}

机构：

[1] Bambino Gesu Childrens Res Hosp, Unit Neuromuscular & Neurodegenerat Disorders, Piazza St Onofrio 4, I-00165 Rome, Italy

[2] Policlin A Gemelli, Ctr Clin Nemo, Largo A Gemelli 8, I-00168 Rome, Italy

[3] Univ Cattolica Sacro Cuore, Paediat Neurol Unit, Largo A Gemelli 8, I-00168 Rome, Italy

来源：

NATURE REVIEWS NEUROLOGY | 2018年 / 14卷 / 04期

关键词：

D O I：

10.1038/nrneurol.2017.189

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Effective drug development for infantile-onset spinal muscular atrophy (SMA) requires a meaningful understanding of disease progression and reliable biomarkers. A new report presents the results of a longitudinal, multicentre, prospective natural history study of SMA, which are critical for the research of future therapies.

引用

页码：197 / 198

页数：2

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