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

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

Research output: Contribution to journal › Review article › peer-review

50 Scopus citations

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.

Original language	English (US)
Pages (from-to)	49-65
Number of pages	17
Journal	Neurodegenerative disease management
Volume	6
Issue number	1
DOIs	https://doi.org/10.2217/nmt.15.73
State	Published - Jan 19 2016

Keywords

antioxidants
erythropoietin
FRDA
Friedreich's ataxia
HDAC
iron

ASJC Scopus subject areas

Medicine(all)

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10.2217/nmt.15.73

Cite this

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title = "Emerging therapies in Friedreich's ataxia",

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 = "antioxidants, erythropoietin, FRDA, Friedreich's ataxia, HDAC, iron",

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AU - Kuo, Sheng Han

AU - Fogel, Brent L.

AU - Wilmot, George R.

AU - Perlman, Susan L.

AU - Onyike, Chiadi U.

AU - Ying, Sarah H.

AU - Zesiewicz, Theresa A.

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Y1 - 2016/1/19

N2 - 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.

AB - 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.

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Emerging therapies in Friedreich's ataxia

Abstract

Keywords

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