Gaboxadol

Unassigned

New Medicines

Angelman syndrome

Information

New molecular entity
Ovid Therapeutics
Ovid Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Delta-selective GABAA receptor agonist - thought to impact neuronal activity by restoring levels of tonic inhibition that exist when intrinsic GABAA levels are low. [1,2]
Prevalence is estimated to be ~1 in 12,000-20,000 people in the general population, affecting males and females equally, but many go undiagnosed. Angelman Syndrome is a genetic disorder mostly affecting the nervous system. It is marked by developmental delay, intellectual disability, severe speech impairment and movement and balance problems. [3,6]
Angelman syndrome
Oral