Blog
N-Acetyl-leucine in progressive CACNA1A ataxia: A case series
CACNA1A-related disorders are rare and progressive; to date, there is no approved treatment. Trials with N-acetyl-leucine (NALL) demonstrated efficacy in disorders featuring ataxia, cognitive impairment, and epilepsy. Accordingly, we hypothesized that NALL may be effective in CACNA1A-associated disorders.
Update on the pharmacotherapy of cerebellar and central vestibular disorders
Modern pharmacotherapy for central vestibular syndromes and cerebellar disorders is actively evolving and includes new medications aimed at improving coordination, reducing nystagmus, and normalizing cerebellar function. Some have already shown effectiveness in clinical studies, while new therapies continue to be investigated in international trials.
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.