Original Investigation

Efficacy and Safety of Ecopipam for Tourette Syndrome

JAMA Neurology 10.1001/jamaneurol.2026.1431

May 26, 2026 at 11:00 AM EDT

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Is ecopipam well tolerated and efficacious in children, adolescents, and adults with Tourette syndrome?This randomized clinical trial included a 12-week open-label period and a 12-week double-blind, randomized withdrawal period for individuals with clinically meaningful tic reduction. Ecopipam was well tolerated and significantly reduced the probability of relapse in pediatric participants.Ecopipam induced and maintained a clinically meaningful reduction in severity of tics for up to 24 weeks.

Corresponding Author: Donald L. Gilbert, MD, MS, Division of Neurology, Cincinnati Children’s Hospital Medical Center, 3333 Burnet Ave, Cincinnati, OH 45229 (donald.gilbert@cchmc.org).

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