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脊肌萎缩症治疗研究进展

刘宇,李洪

Modern Journal of Integrated Traditional Chinese and Western Medicine（2010）

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Abstract
脊肌萎缩症(SMA)是一组由于下运动神经元变性导致进行性无力和肌萎缩的疾病,是婴幼儿期最常见的致死性常染色体隐性遗传病,在中国南方的发病率为1/53000[1],一直无特效治疗.近十余年来分子遗传学上的突破,给本病的治疗带来曙光.笔者就国内外对SMA治疗上的研究进展作一综述.
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