Emerging therapies in Friedreich's ataxia

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.

PMID: 26782317
PMCID: PMC4768799
DOI: 10.2217/nmt.15.73

Review

Emerging therapies in Friedreich's ataxia

Tanya V Aranca et al. Neurodegener Dis Manag. 2016.

. 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.

PMID: 26782317
PMCID: PMC4768799
DOI: 10.2217/nmt.15.73

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.

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Conflict of interest statement

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

References

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Emerging therapies in Friedreich's ataxia

Affiliations

Emerging therapies in Friedreich's ataxia

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Conflict of interest statement

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