The results from the phase 2b randomized clinical trial, published November 20th in Annals of Neurology, build on more than a decade of research spurred by anecdotal reports from patients with LD who said their symptoms improved after consuming a couple alcoholic drinks.

In the trial of more than 100 patients, a single dose of sodium oxybate significantly improved symptoms of patients with alcohol-responsive LD without causing serious side effects.

The minimum efficacy of the drug was 16% of voice improvement, with the average of 41% in patients with alcohol-responsive LD.

Sodium oxybate showed no significant changes from placebo in LD patients whose symptoms do not improve with alcohol.

In previous open label trials, Simonyan's team showed that sodium oxybate improves voice symptoms in 82% of patients with alcohol-responsive LD.