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.
May 11th 2024
A recent study recognized anti-DAGLA autoantibodies in the cerebrospinal fluid of a small group of patients, suggesting that it could be a potential biomarker for diagnosing cerebellar ataxia.
SARA Scale Scores Strongly Predictive of Friedreich Ataxia Progression
October 1st 2023Over 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.
Phase 2 Trial Shows Increased Frataxin Levels in Friedreich Ataxia Following CT-1601 Treatment
August 3rd 2023Positive 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.
Patient Dosing Complete for Part 1 of Gene Therapy Trial in Friedreich Ataxia Cardiomyopathy
July 20th 2023The 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.
Biohaven Submits New Drug Application for Troriluzole as Spinocerebellar Ataxia Type 3 Therapy
June 25th 2023In 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.