Emerging therapies in hereditary ataxias

Eisel MLS, Burns M, Ashizawa T, Byrne B, Corti M, Subramony SH. Emerging therapies in hereditary ataxias. Trends Mol Med. August 16, 2024 ;0(0). doi:10.1016/j.molmed.2024.07.008 

In this review, we focus on Friedreich ataxia (FRDA) and the polyglutamine ataxias in which translational research is active. However, much remains to be done to identify safe and effective molecules, create ideal delivery methods, and perform innovative clinical trials to prove the safety and efficacy of treatments for these rare but devastating diseases.