Ataxia

Over a 4-year follow-up, SARA progression was substantially linear. Age at evaluation also influenced the speed of SARA progression, while disease duration did not improve the prediction of the statistical model.

Over a 3-treatment cycle, no new clinically significant safety observations were found, with significant dose-dependent increases in frataxin in muscle mRNA expression.

Positive data from the 25 mg cohort study exploring Larimar Therapeutics’ CTI-1601 has been submitted to the FDA, with a meeting scheduled with the agency later this quarter to discuss steps for a phase 2 trial.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is ataxia.

The 52-week, dose-ascending trial will have data read out in the first half of 2024, with long-term safety and efficacy evaluated over a 5-year period following completion of the initial trial.

In a phase 3 study, a subgroup of patients with spinocerebellar ataxia treated with troriluzole demonstrated a numerical treatment benefit relative to placebo on the primary end point of change in f-SARA scores.

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.