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 Hung Ying, Theresa A. Zesiewicz

Research output: Contribution to journalReview articlepeer-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.

Original languageEnglish (US)
Pages (from-to)49-65
Number of pages17
JournalNeurodegenerative disease management
Volume6
Issue number1
DOIs
StatePublished - 2016

Keywords

  • FRDA
  • Friedreich's ataxia
  • HDAC
  • antioxidants
  • erythropoietin
  • iron

ASJC Scopus subject areas

  • Medicine(all)

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