Tourette syndrome: effects of acetyl-leucine in two individuals
Тourette Syndrome (TS) is a childhood-onset neurodevelopmental disorder marked by involuntary motor and vocal tics, often treated with behavioral therapies or antipsychotics that can have limited accessibility or side effects. Acetyl-leucine (AL), a modified amino acid recently approved for Niemann-Pick disease type C, has shown potential in improving neurological symptoms in various disorders, prompting its off-label use in two adult TS patients. Both individuals experienced a clinically meaningful reduction in tic severity, obsessive–compulsive symptoms, and improved quality of life after six weeks of AL treatment at 5 g/day, with sustained benefits observed six weeks post-treatment. Although limited by a small sample and open-label design, these promising results support the need for further placebo-controlled trials to evaluate AL as a treatment option for TS.
