正在开发的治疗弗里德里希共济失调的孤儿药:重点关注奥那韦洛龙

IF 2.1 Q3 CLINICAL NEUROLOGY Degenerative neurological and neuromuscular disease Pub Date : 2019-10-15 DOI:10.2147/DNND.S180027
Shaila D. Ghanekar, W. W. Miller, C. Meyer, Kevin J Fenelon, Alvin Lacdao, T. Zesiewicz
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引用次数: 3

摘要

摘要弗里德里希共济失调(FRDA)是一种破坏性的进行性共济失调,其特征是线粒体功能障碍和氧化应激。Nrf2激活剂如奥马韦洛龙(Omav)调节抗氧化机制,因此可能是FRDA中可行的治疗剂。
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Orphan Drugs In Development For The Treatment Of Friedreich’s Ataxia: Focus On Omaveloxolone
Abstract Friedreich’s Ataxia (FRDA) is a devastating and progressive ataxia, marked by mitochondrial dysfunction and oxidative stress. Nrf2 activators such as omaveloxolone (Omav) modulate antioxidative mechanisms, and thus may be viable therapeutic agents in FRDA.
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