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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
The New England Journal of Medicine ( IF 158.5 ) Pub Date : 2017-11-01 , DOI: 10.1056/nejmoa1702752 Richard S. Finkel , Eugenio Mercuri , Basil T. Darras , Anne M. Connolly , Nancy L. Kuntz , Janbernd Kirschner , Claudia A. Chiriboga , Kayoko Saito , Laurent Servais , Eduardo Tizzano , Haluk Topaloglu , Már Tulinius , Jacqueline Montes , Allan M. Glanzman , Kathie Bishop , Z. John Zhong , Sarah Gheuens , C. Frank Bennett , Eugene Schneider , Wildon Farwell , Darryl C. De Vivo
The New England Journal of Medicine ( IF 158.5 ) Pub Date : 2017-11-01 , DOI: 10.1056/nejmoa1702752 Richard S. Finkel , Eugenio Mercuri , Basil T. Darras , Anne M. Connolly , Nancy L. Kuntz , Janbernd Kirschner , Claudia A. Chiriboga , Kayoko Saito , Laurent Servais , Eduardo Tizzano , Haluk Topaloglu , Már Tulinius , Jacqueline Montes , Allan M. Glanzman , Kathie Bishop , Z. John Zhong , Sarah Gheuens , C. Frank Bennett , Eugene Schneider , Wildon Farwell , Darryl C. De Vivo
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Among infants with spinal muscular atrophy, those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group. Early treatment may be necessary to maximize the benefit of the drug. (Funded by Biogen and Ionis Pharmaceuticals; ENDEAR ClinicalTrials.gov number, NCT02193074.)
中文翻译：
Nusinersen与假手术控制小儿发作性脊髓性肌萎缩症的关系
在患有脊髓性肌萎缩症的婴儿中，接受神经营养素治疗的婴儿比对照组的婴儿更有生命，并且运动功能得到改善。为了使药物的益处最大化，可能必须进行早期治疗。（由Biogen和Ionis Pharmaceuticals资助； ENDEAR ClinicalTrials.gov编号，NCT02193074。）
更新日期：2017-11-02
中文翻译：
Nusinersen与假手术控制小儿发作性脊髓性肌萎缩症的关系
在患有脊髓性肌萎缩症的婴儿中，接受神经营养素治疗的婴儿比对照组的婴儿更有生命，并且运动功能得到改善。为了使药物的益处最大化，可能必须进行早期治疗。（由Biogen和Ionis Pharmaceuticals资助； ENDEAR ClinicalTrials.gov编号，NCT02193074。）
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