New developments in pharmacotherapy for Friedreich ataxia

Alexandra Clay, Patrick Hearle, Kim Schadt & David R. Lynch (2019) New developments in pharmacotherapy for Friedreich ataxia, Expert Opinion on Pharmacotherapy, DOI: 10.1080/14656566.2019.1639671

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.


New developments in pharmacotherapy for Friedreich ataxia