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.
