SPOTLIGHT -
The NeurologyLive® Ataxia Disease Spotlight page provides in-depth updates and specific coverage on the latest data readouts and expert conversations related to the treatment and management of patients with the movement disorder.
Vatiquinone Shows Additional Benefits on Friedreich Ataxia Disease Progression Despite Failing to Meet Primary End Point
Over a 72-week treatment period, vatiquinone showed significant impacts on bulbar and upright stability subscales of the modified Friedreich Ataxia Rating Scale score.
Dosing Commenced in Phase 1/2a Trial of VO659 in Huntington Disease and Spinocerebellar Ataxia Types 1 and 3
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.
Impaired Decision-Making Symptom Significantly Associated in Cerebellar Ataxia
Patients with cerebellar ataxia made riskier decisions than controls, suggesting this may be a unique cerebellar cognitive symptom.
Activation of NRF2 and FXN in Friedreich Ataxia
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.
FDA Approves Omaveloxolone As First Treatment for Friedreich Ataxia
The Reata Pharmaceuticals therapy, branded as Skyclarys, is indicated for adults and adolescents aged 16 years and older.