Emerging therapies in Friedreich's ataxia

Author:

Aranca Tanya V1,Jones Tracy M1,Shaw Jessica D1,Staffetti Joseph S1,Ashizawa Tetsuo2,Kuo Sheng-Han3,Fogel Brent L4,Wilmot George R5,Perlman Susan L6,Onyike Chiadi U7,Ying Sarah H8910,Zesiewicz Theresa A111

Affiliation:

1. University of South Florida Ataxia Research Center, Department of Neurology, FL, USA

2. McKnight Brain Institute, University of Florida Department of Neurology, FL, USA

3. Department of Neurology, Columbia University, NY, USA

4. Department of Neurology, Neurogenetics Program, David Geffen School of Medicine, University of California, CA, USA

5. Department of Neurology, Emory University, GA, USA

6. Ataxia and Huntington Disease Center of Excellence, University of California, CA, US

7. Department of Psychiatry and Behavioral Sciences, Johns Hopkins University school of Medicine MD, USA

8. Department of Neurology, Johns Hopkins University School of Medicine, MD, USA

9. Department of Radiology and Radiological Sciences, Johns Hopkins University School of Medicine, MD, USA

10. Department of Ophthalmology, Johns Hopkins University School of Medicine, MD, USA

11. James A. Haley Veterans’ Hospital, FL, USA

Abstract

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.

Publisher

Future Medicine Ltd

Subject

Neurology (clinical)

Cited by 59 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3