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Author Notes:

Author for correspondence: tzesiewi@health.usf.edu

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.

Subjects:

Keywords:

  • FRDA
  • Friedreich's ataxia
  • HDAC
  • antioxidants
  • erythropoietin
  • iron
  • Adolescent
  • Clinical Trials as Topic
  • Drug Therapy, Combination
  • Friedreich Ataxia
  • Genetic Therapy
  • Humans
  • Nerve Growth Factors
  • Severity of Illness Index
  • Treatment Outcome

Emerging therapies in Friedreich's ataxia.

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Tools:

Journal Title:

Neurodegenerative Disease Management

Volume:

Volume 6, Number 1

Publisher:

, Pages 49-65

Type of Work:

Article | Post-print: After Peer Review

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.
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