Emerging therapies in Friedreich's ataxia.
Emerging therapies in Friedreich's ataxia.
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DOI：
10.2217/nmt.15.73
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发表时间：
2016
影响因子：
2.6
通讯作者：
Zesiewicz TA
中科院分区：
文献类型：
--
作者：
Aranca TV;Jones TM;Shaw JD;Staffetti JS;Ashizawa T;Kuo SH;Fogel BL;Wilmot GR;Perlman SL;Onyike CU;Ying SH;Zesiewicz TA
弗里德赖希共济失调（FRDA）是一种遗传性、进行性神经退行性疾病，通常影响青少年和年轻人。近年来，治疗策略和疾病洞察力迅速扩大，为FRDA人群带来了希望。目前还没有美国FDA批准的FRDA治疗方法，但其发病机制的研究进展已经导致了潜在治疗方法的临床试验。本文回顾了新兴的治疗方法，并讨论了未来的前景，包括需要更精确的措施来检测神经系统症状的变化以及疾病修饰剂。
Friedreich’s ataxia (FRDA) is an inherited, progressive neurodegenerative disease that typically affects teenagers and young adults. Therapeutic strategies and disease insight have expanded rapidly over recent years, leading to hope for the FRDA population. There is currently no US FDA-approved treatment for FRDA, but advances in research of its pathogenesis have led to clinical trials of potential treatments. This article reviews emerging therapies and discusses future perspectives, including the need for more precise measures for detecting changes in neurologic symptoms as well as a disease-modifying agent.