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Duchenne肌营养不良症：辉瑞基因疗法的早期数据

2019-06-29 Allan MedSci原创

Duchenne肌营养不良症基因疗法儿科PF-06939926
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辉瑞公司近日公布了PF-06939926的初始Ib期研究数据，结果显示实验性基因疗法导致男性Duchenne肌营养不良症（DMD）中微小肌营养不良蛋白表达增加。

辉瑞公司近日公布了PF-06939926的初始Ib期研究数据，结果显示实验性基因疗法导致男性Duchenne肌营养不良症（DMD）中微小肌营养不良蛋白表达增加。该试验的主要终点是安全性和耐受性，次要终点是肌纤维内微小肌营养不良蛋白的表达。结果显示，在接受PF-06939926的患者中，微小肌营养不良蛋白的平均表达水平为23.6％。

Duchenne肌营养不良症是一种X染色体隐性遗传疾病，主要发生于男孩。全球平均每3500个新生男婴中就有一人罹患此病。患者一般在3-5岁开始发病，最早表现出进行性腿部肌无力，导致不便行走。12岁时失去行走能力，20岁-30岁时会因呼吸衰竭而死亡。


原始出处：

http://www.firstwordpharma.com/node/1650348#axzz5s0nRyue8

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    2019-08-07 yilong5287542

    #Duchenne肌营养不良症#

    0

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    2019-07-19 ms4468361851264128

    #肌营养不良症#

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    2019-10-26 fmq_28614553

    #Duchenne肌营养不良#

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    2019-12-28 makuansheng

    #Chen#

    0

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    2020-04-26 751943081_32627960

    #肌营养不良#

    0

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    2019-06-29 坚强007

    向挑战病魔的科研人员致敬！

    0

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    2019-06-29 飛歌

    学习了很有用不错

    0

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