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Expert Opin Pharmacother. 2019 Jul 16:1-13. doi: 10.1080/14656566.2019.1639671. [Epub ahead of print]

New developments in pharmacotherapy for Friedreich ataxia.

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a Division of Neurology , Children's Hospital of Philadelphia , Philadelphia , PA , USA.


Introduction: Friedreich ataxia (FRDA), a rare disease caused by the deficiency of the mitochondrial matrix protein frataxin, affects roughly 1 in 50,000 individuals worldwide. Current and emerging therapies focus on reversing the deleterious effects of such deficiency including mitochondrial augmentation and increasing frataxin levels, providing the possibility of treatment options for this physiologically complex, multisystem disorder. Areas covered: In this review article, the authors discuss the current and prior in vivo and in vitro research studies related to the treatment of FRDA, with a particular interest in future implications of each therapy. Expert opinion: Since the discovery of FXN in 1996, multiple clinical trials have occurred or are currently occurring; at a rapid pace for a rare disease. These trials have been directed at the augmentation of mitochondrial function and/or alleviation of symptoms and are not regarded as potential cures in FRDA. Either a combination of therapies or a drug that replaces or increases the pathologically low levels of frataxin better represent potential cures in FRDA.


EPI-743; Friedreich ataxia; HDAC inhibitors; Nrf2 activators; coenzyme Q10; idebenone; interferon gamma; tat-frataxin

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