Monday, Stoke Therapeutics Inc (NASDAQ:STOK) released new data from two open-label Phase 1/2a studies and two open-label extension (OLE) studies of children and adolescents ages 2 to 18 with Dravet syndrome who were treated with STK-001.
Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged, and refractory seizures.
Data showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior.
STK-001 has been generally well-tolerated in studies to date.
Patients enrolled in these studies were highly refractory to treatment and taking the best available anti-seizure medicines: 85% of patients were taking at ...