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

40 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 - 2016
Externally published	Yes

Keywords

FRDA
Friedreich's ataxia
HDAC
antioxidants
erythropoietin
iron

ASJC Scopus subject areas

General Medicine

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10.2217/nmt.15.73

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

author = "Aranca, {Tanya V.} and Jones, {Tracy M.} and Shaw, {Jessica D.} and Staffetti, {Joseph S.} and Tetsuo Ashizawa and Kuo, {Sheng Han} and Fogel, {Brent L.} and Wilmot, {George R.} and Perlman, {Susan L.} and Onyike, {Chiadi U.} and Ying, {Sarah H.} and Zesiewicz, {Theresa A.}",

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doi = "10.2217/nmt.15.73",

language = "English (US)",

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AU - Aranca, Tanya V.

AU - Jones, Tracy M.

AU - Shaw, Jessica D.

AU - Staffetti, Joseph S.

AU - Ashizawa, Tetsuo

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.

PY - 2016

Y1 - 2016

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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KW - Friedreich's ataxia

KW - HDAC

KW - antioxidants

KW - erythropoietin

KW - iron

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Emerging therapies in Friedreich's ataxia

Abstract

Keywords

ASJC Scopus subject areas

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