Friedreich ataxia- pathogenesis and implications for therapies

Martin B. Delatycki, Sanjay I. Bidichandani, Neurobiology of Disease, 2019, 104606, doi: 10.1016/j.nbd.2019.104606.

Therapies that have been proposed include pharmaceuticals that increase frataxin levels, protein and gene replacement therapies, antioxidants, iron chelators and modulators of inflammation. Whilst 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.

Friedreich ataxia- pathogenesis and implications for therapies