Myoclonic Dystonia

WEDNESDAY, Nov. 27, 2024 -- Sodium oxybate improves symptoms of alcohol-responsive (EtOH-positive) isolated laryngeal dystonia (LD), according to a study published online Nov. 20 in the Annals of Neurology . Kristina Simonyan, M.D., Ph.D., from Massachusetts Eye and Ear and Harvard Medical School in Boston, and colleagues examined the efficacy and safety of sodium oxybate versus placebo in a phase 2b randomized, crossover clinical trial involving patients with isolated LD. The study was conducted in 106 patients with EtOH-positive and alcohol-non-responsive (EtOH-negative) LD who were randomly assigned to receive 1.5 g sodium oxybate first or matching placebo first (53 each). The change from baseline in LD symptom severity 40 minutes after drug intake was assessed as the primary outcome. The researchers found that EtOH-positive but not EtOH-negative patients had a significant improvement in LD symptoms following sodium oxybate versus placebo compared with baseline. In EtOH-positive patients, statistically significant minimum drug efficacy was found at ≥16 percent symptom improvement (odds ratio, 2.09), with an average benefit of 40.81 percent. By 300 minutes after intake, drug efficacy waned, without a rebound. There were no changes in cognitive function, suicide, or vital signs. Mild dizziness, nausea, and daytime sleepiness were common adverse events. "Our findings suggest that sodium oxybate can be taken on an as-needed basis, such as before work or a social event, so patients can tailor treatment to their own daily needs and get in control of their symptoms," Simonyan said in a statement. Abstract/Full Text Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.