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研究性基因疗法SRP-9003可改善肢带肌营养不良症患者的功能

2019-10-05 Allan MedSci原创

SRP-9003基因疗法肢带肌营养不良症
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Sarepta Therapeutics公司近日公布了I / IIa期试验的最新结果，该结果显示接受研究性基因疗法SRP-9003的三名2E型肢带型肌肉营养不良（LGMD2E）患者的功能得到了改善。在治疗9个月时，观察到所有功能结局都有改善，包括NSAD以及10米和100米步行测试。

Sarepta Therapeutics公司近日公布了I / IIa期试验的最新结果，该结果显示接受研究性基因疗法SRP-9003的三名2E型肢带型肌肉营养不良（LGMD2E）患者的功能得到了改善。在治疗9个月时，观察到所有功能结局都有改善，包括NSAD以及10米和100米步行测试。Sarepta还报告说，与基线相比，平均肌酸激酶水平在9个月时明显降低。此外，该公司还补充说，没有观察到新的安全信号。根据Sarepta的说法，SRP-9003旨在编码全长天然β-糖聚糖蛋白，而缺乏该基因是LGMD2E的唯一原因。

LGMD的定义是肌无力主要呈近端分布的肌营养不良。该病包括一些异质性遗传病，后者在严重程度、表型、病理学及发病年龄（从儿童期至成年期）方面存在不同。目前LGMD仍作为描述呈带状分布的肌营养不良患者的通用术语。


原始出处：

http://www.firstwordpharma.com/node/1671751#axzz61Rlned4y

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    2019-11-10 jeanqiuqiu

    #SRP-9003#

    0

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    2019-10-22 751943081_32627960

    #肌营养不良#

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    2019-12-08 ms4468361851264128

    #肌营养不良症#

    0

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    2019-10-07 thm112988

    好

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    2019-10-07 1e1c07bdm01(暂无匿称)

    好

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    2019-10-06 坚强007

    向科研人员致敬！

    0