Levacetylleucine for AT
2026In this first positive phase 3 trial in ataxia-telangiectasia, levacetylleucine produced a statistically significant and clinically meaningful improvement in SARA (treatment effect −1·88 points, 95...
Does ecopipam, a selective dopamine D1 receptor antagonist, maintain tic improvement and remain well tolerated for up to 24 weeks in patients with Tourette syndrome?
Ecopipam TS Phase 3
Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial
JAMA Neurology · 2026
Bottom Line: Ecopipam 1.8 mg/kg/day significantly reduced the risk of relapse and maintained clinically meaningful tic reduction over 24 weeks in pediatric patients with Tourette syndrome, with adverse events primarily affecting the central nervous system and no clinically meaningful effects on weight, metabolic parameters, or drug-induced movement disorders.