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Progress in the treatment of Friedreich ataxia
Affiliations- Bruce Lefroy Centre for Genetic Health Research, Murdoch Children's Research Institute, Parkville, Australia
- School of Psychological Science, Faculty of Medicine, Nursing and Health Sciences, Monash University, Clayton, Australia
- Department of Paediatrics, University of Melbourne, Parkville, Australia
- Department of Neurology, Royal Children's Hospital, Parkville, Australia
- Victorian Clinical Genetics Services, Parkville, Australia
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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.
Keywords
Friedreich ataxia, Therapy, Rehabilitation, Gene therapy
About this articleTitle
Progress in the treatment of Friedreich ataxia
Journal
Neurologia i Neurochirurgia Polska
Issue
Article type
Review Article
Pages
129-139
Published online
2018-02-19
Page views
1698
Article views/downloads
1995
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
