open access

Vol 52, No 2 (2018)
Review article
Submitted: 2018-01-30
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Progress in the treatment of Friedreich ataxia

Geneieve Tai, Louise A. Corben, Eppie M. Yiu, Sarah C. Milne, Martin B. Delatycki
DOI: 10.1016/j.pjnns.2018.02.003
·
Neurol Neurochir Pol 2018;52(2):129-139.

open access

Vol 52, No 2 (2018)
Review article
Submitted: 2018-01-30

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.

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.

Get Citation

Keywords

Friedreich ataxia, Therapy, Rehabilitation, Gene therapy

About this article
Title

Progress in the treatment of Friedreich ataxia

Journal

Neurologia i Neurochirurgia Polska

Issue

Vol 52, No 2 (2018)

Pages

129-139

DOI

10.1016/j.pjnns.2018.02.003

Bibliographic record

Neurol Neurochir Pol 2018;52(2):129-139.

Keywords

Friedreich ataxia
Therapy
Rehabilitation
Gene therapy

Authors

Geneieve Tai
Louise A. Corben
Eppie M. Yiu
Sarah C. Milne
Martin B. Delatycki

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