Disease Spotlight: Ataxia

Over a 72-week treatment period, vatiquinone showed significant impacts on bulbar and upright stability subscales of the modified Friedreich Ataxia Rating Scale score.

VO659, the only clinical candidate targeting the CAG repeat expansion that causes all polyglutamine diseases, is designed to reduce mutant HTT and spare wildtype HTT.

Patients with cerebellar ataxia made riskier decisions than controls, suggesting this may be a unique cerebellar cognitive symptom.

Disease pathogenesis is attributed to oxidative stress—which can be regulated by NRF2, which, in turn, binds to antioxidant responsive elements in the promoter of the target gene FXN to control its expression.

The Reata Pharmaceuticals therapy, branded as Skyclarys, is indicated for adults and adolescents aged 16 years and older.