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Friedreich ataxia—update on pathogenesis and possible therapies

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Abstract.

Friedreich ataxia is the most-common inherited ataxia. Since the causative genetic basis was described in 1996, much has been learnt about the pathogenesis from human, animal, and yeast studies. This has led to the development of rational therapeutic approaches. In this review, the current state of knowledge regarding the pathogenesis of Friedreich ataxia is presented and possible therapeutic strategies based on this knowledge are discussed.

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Authors and Affiliations

  1. Department of Cellular Animal Physiology, Subfaculty of Biology, Faculty of Science, University of Nijmegen, Nijmegen, The Netherlands

    Max Voncken

  2. Murdoch Childrens Research Institute, Royal Children’s Hospital, Victoria 3052, Parkville, Australia

    Max Voncken, Panos Ioannou & Martin B. Delatycki

  3. Department of Pediatrics, University of Melbourne, Royal Children’s Hospital, Victoria 3052, Parkville, Australia

    Panos Ioannou & Martin B. Delatycki

  4. Bruce Lefroy Centre for Genetic Health Research, Genetic Health Services Victoria, Royal Children’s Hospital, Flemington Road, Victoria 3052, Parkville, Australia

    Martin B. Delatycki

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  1. Max Voncken
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  2. Panos Ioannou
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  3. Martin B. Delatycki
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Correspondence to Martin B. Delatycki.

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Voncken, M., Ioannou, P. & Delatycki, M.B. Friedreich ataxia—update on pathogenesis and possible therapies. Neurogenetics 5, 1–8 (2004). https://doi.org/10.1007/s10048-003-0170-z

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