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FARAFARA Cure FA

Friedreich ataxia- pathogenesis and implications for therapies

This is a review article focusing on the insights into the pathogenesis of this disorder and how those insights are being translated into novel therapeutic approaches. For example, more recently, a role for inflammation has emerged as being important in the pathogenesis of Friedreich ataxia. These findings have led to a number of potential therapies that have been subjected to clinical trials or are being developed toward human studies. Therapies that have been proposed include pharmaceuticals that increase frataxin levels, protein and gene replacement therapies, antioxidants, iron chelators and modulators of inflammation. While no therapies have yet been approved for Friedreich ataxia, there is much optimism that the advances in the understanding of the pathogenesis of this disorder since the discovery its genetic basis, will result in approved disease modifying therapies in the near future.

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About the Author

Jen Farmer

Jen Farmer

Executive Director

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