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COMPOSITIONS AND METHODS FOR THE PREVENTION AND/OR TREATMENT OF MITOCHONDRIAL DISEASE, INCLUDING FRIEDREICH'S ATAXIA

机译:用于预防和/或治疗线粒体疾病的组合物和方法,包括Friedreich的共济失调

摘要

The disclosure provides therapeutic compositions (i.e., therapeutic agents) and methods of preventing or treating Friedreich's ataxia in a mammalian subject, reducing risk factors, signs and/or symptoms associated with Friedreich's ataxia (e.g., Complex I deficiency), and/or reducing the likelihood or severity of Friedreich's ataxia. The disclosure further provides novel intermediates for the production of said therapeutic compositions and related reduced versions of said therapeutic compositions, which reduce forms may also be used as therapeutic agents (or prodrugs of the therapeutic agent(s)).
机译:本公开内容提供治疗组合物(即治疗剂)和预防或治疗哺乳动物受试者的共济失调的方法,减少与Friedreich共济失调(例如,复杂的I缺乏)相关的危险因素,征兆和/或症状,和/或减少弗里德雷希共济失调的可能性或严重程度。本发明进一步提供了用于生产所述治疗组合物的新型中间体,以及所述治疗组合物的相关简化版本,其减少形式也可用作治疗剂(或治疗剂的前药)。

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