Emerging therapies in Friedreich's ataxia

Tanya V Aranca; Tracy M Jones; Jessica D Shaw; Joseph S Staffetti; Tetsuo Ashizawa; Sheng-Han Kuo; Brent L Fogel; George R Wilmot; Susan L Perlman; Chiadi U Onyike; Sarah H Ying; Theresa A Zesiewicz

doi:10.2217/nmt.15.73

Emerging therapies in Friedreich's ataxia

Neurodegener Dis Manag. 2016;6(1):49-65. doi: 10.2217/nmt.15.73.

Authors

Affiliations

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.

Keywords: FRDA; Friedreich's ataxia; HDAC; antioxidants; erythropoietin; iron.

Publication types

Review

MeSH terms

Adolescent
Clinical Trials as Topic
Drug Therapy, Combination / trends
Friedreich Ataxia / genetics
Friedreich Ataxia / rehabilitation
Friedreich Ataxia / therapy*
Genetic Therapy / trends
Humans
Nerve Growth Factors / therapeutic use
Severity of Illness Index
Treatment Outcome

Substances

Nerve Growth Factors

Abstract

Publication types

MeSH terms

Substances

Grants and funding