Progress in the treatment of Friedreich ataxia

Research output: Contribution to journalReview ArticleResearchpeer-review

Abstract

Friedreich ataxia (FRDA) is a progressive neurological disorder affecting approximately 1 in 29,000 individuals of European descent. At present, there is no approved pharmacological treatment for this condition however research into treatment of FRDA has advanced considerably over the last two decades since the genetic cause was identified. Current proposed treatment strategies include decreasing oxidative stress, increasing cellular frataxin, improving mitochondrial function as well as modulating frataxin controlled metabolic pathways. Genetic and cell based therapies also hold great promise. Finally, physical therapies are being explored as a means of maximising function in those affected by FRDA.

Original languageEnglish
Pages (from-to)129-139
Number of pages11
JournalNeurologia i Neurochirurgia Polska
Volume52
Issue number2
DOIs
Publication statusPublished - 1 Mar 2018

Keywords

  • Friedreich ataxia
  • Gene therapy
  • Rehabilitation
  • Therapy

Cite this

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title = "Progress in the treatment of Friedreich ataxia",
abstract = "Friedreich ataxia (FRDA) is a progressive neurological disorder affecting approximately 1 in 29,000 individuals of European descent. At present, there is no approved pharmacological treatment for this condition however research into treatment of FRDA has advanced considerably over the last two decades since the genetic cause was identified. Current proposed treatment strategies include decreasing oxidative stress, increasing cellular frataxin, improving mitochondrial function as well as modulating frataxin controlled metabolic pathways. Genetic and cell based therapies also hold great promise. Finally, physical therapies are being explored as a means of maximising function in those affected by FRDA.",
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author = "Geneieve Tai and Corben, {Louise A.} and Yiu, {Eppie M.} and Milne, {Sarah C.} and Delatycki, {Martin B.}",
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Progress in the treatment of Friedreich ataxia. / Tai, Geneieve; Corben, Louise A.; Yiu, Eppie M.; Milne, Sarah C.; Delatycki, Martin B.

In: Neurologia i Neurochirurgia Polska, Vol. 52, No. 2, 01.03.2018, p. 129-139.

Research output: Contribution to journalReview ArticleResearchpeer-review

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