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Drug Development for the Therapy of Mitochondrial Diseases.
Trends in Molecular Medicine ( IF 13.6 ) Pub Date : 2019-11-11 , DOI: 10.1016/j.molmed.2019.09.002
Volkmar Weissig 1
Affiliation  

Mitochondrial diseases are a heterogeneous group of inherited or acquired devastating disorders that affect the energy metabolism of the body. Many strategies have been investigated, but currently there is no FDA-approved drug that can alleviate disease symptoms or slow disease progression. This review analyzes to what extent growing knowledge over the past two decades about the etiology and pathogenesis of mitochondrial diseases is reflected in the design and development of new experimental drugs for the therapy of these disorders. All currently registered clinical trials involving new experimental drug entities are reviewed to evaluate how far away we are from the first FDA-approved drug therapy for mitochondrial disease.

中文翻译:

用于线粒体疾病治疗的药物开发。

线粒体疾病是影响身体能量代谢的遗传性或获得性破坏性疾病的异质性群体。已经研究了许多策略,但是目前尚无FDA批准的可减轻疾病症状或减慢疾病进展的药物。这篇综述分析了过去二十年来关于线粒体疾病的病因和发病机理的日益增长的知识在设计和开发用于治疗这些疾病的新实验药物中得到了反映。审查所有涉及新的实验性药物实体的当前注册临床试验,以评估我们与首批FDA批准的线粒体疾病药物疗法之间的距离。
更新日期:2020-01-29
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