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mNIS+7 and lower limb function in inotersen treatment of hereditary transthyretin-mediated amyloidosis.
Muscle & Nerve ( IF 3.4 ) Pub Date : 2020-07-12 , DOI: 10.1002/mus.27022
P James B Dyck 1 , John C Kincaid 2 , Janice F Wiesman 3 , Michael Polydefkis 4 , William J Litchy 1 , Michelle L Mauermann 1 , Elizabeth J Ackermann 5 , Spencer Guthrie 6 , Michael Pollock 7 , Shiangtung W Jung 8 , Brenda F Baker 8 , Peter J Dyck 1
Affiliation  

Inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, demonstrated significant benefit versus placebo in the modified Neuropathy Impairment Score (NIS) +7 neurophysiologic tests (mNIS+7) in patients with hereditary TTR‐mediated amyloidosis (hATTR) with polyneuropathy. This analysis assessed the mNIS+7 components by anatomic location and the lower limb function (LLF) test.

中文翻译:

在肌钙蛋白治疗遗传性甲状腺素介导的淀粉样变性中,mNIS + 7和下肢功能。

在遗传性TTR介导的淀粉样变性(hATTR)伴有多发性神经病的患者中,转甲状腺素蛋白(TTR)蛋白产生的反义寡核苷酸抑制剂Inotersen在改良的神经病障碍评分(NIS)+7神经生理学测试(mNIS + 7)中显示出与安慰剂相比显着的获益。该分析通过解剖位置和下肢功能(LLF)测试评估了mNIS + 7成分。
更新日期:2020-09-20
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