第24届世界肌肉协会年会:AT132治疗X连锁型肌管肌病的ASPIRO试验取得新阳性数据

2019-10-07 Allan MedSci原创

Audentes是一家领先的基于AAV的基因药物公司,致力于开发针对严重的罕见神经肌肉疾病的创新疗法,Audentes今天宣布了ASPIRO的新阳性数据,该临床试验评估了AT132治疗X连锁型肌管肌病(XLMTM)的有效性和安全性。数据由James J. Dowling博士在第24届世界肌肉协会年会上发表。

Audentes是一家领先的基于AAV的基因药物公司,致力于开发针对严重的罕见神经肌肉疾病的创新疗法,Audentes今天宣布了ASPIRO的新阳性数据,该临床试验评估了AT132治疗X连锁型肌管肌病(XLMTM)的有效性和安全性。数据由James J. Dowling博士在第24届世界肌肉协会年会上发表。

James J. Dowling博士说:“在两个剂量组中接受治疗的患者均显示出呼吸机依赖性的显著降低,这表明AT132有潜力为XLMTM患者和家庭带来变革性收益”。XLMTM是一种主要影响运动所用的肌肉(骨骼肌)的疾病,患者常骨骼脆弱,脊柱弯曲,自主呼吸困难,面部表情生硬,反射缺乏。


原始出处:

http://www.firstwordpharma.com/node/1671995#axzz61Rlned4y

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    2020-05-25 xugumin
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    2019-10-07 坚强007

    向科研人员致敬!

    0