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Angelman Syndrome Treatment Safe, Effective in Exploratory Analyses

Key clinical point: OV101 (gaboxadol), a type A gamma-aminobutyric acid receptor agonist, was safe and well tolerated in a phase 2 trial, and improved clinical outcomes in exploratory analyses.

Major finding: Clinician-rated clinical global impressions of improvement were superior for gaboxadol versus placebo (P = .0006), informing further development of a phase 3 trial.

Study details: An analysis of data for 78 patients with Angelman syndrome who completed the STARS study, a 12-week, randomized, phase 2 trial that compared OV101 with placebo.

Disclosures: Funding for the study came from Ovid Therapeutics. Dr. Kolevzon reported disclosures related to Ovid Therapeutics, as well as Coronis Neurosciences, 5AM Ventures, SEMA4, LabCorp, and AMO Pharma, among others.

Citation:

REPORTING FROM AAN 2019