Author Archives: Patricia Inacio PhD

STK-001 Seen to Reduce Seizures in Children With Severe Dravet

Single and multiple doses of Stoke Therapeutics’ investigational therapy STK-001 were well-tolerated and led to a significant reduction in the frequency of convulsive seizures among children and adolescents with Dravet syndrome in the first three months of follow-up, interim data from the ongoing Phase 1/2a MONARCH trial show.

Anti-seizure Therapy NT102 Wins FDA’s Orphan Drug Status

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Neuroene Therapeutics‘ NT102, a potential anti-seizure therapy for Dravet syndrome. NT102, given orally, has been shown to prevent seizures in several animal models, with “excellent protection” against such attacks in Dravet syndrome, according to Neuroene. Orphan drug…

CAMP4 Buys Global Rights to OPKO Health’s RNA-based Therapeutics

CAMP4 Therapeutics has entered an exclusive worldwide licensing agreement to develop, manufacture, and commercialize OPKO Health’s RNA-based technology as potential disease-modifying treatments for Dravet syndrome. As part of the deal, CAMP4 has prioritized OPKO’s lead candidate for Dravet to enter clinical development. “We are delighted to enter into…

Applications Open for Patient Assistance Grants Program

The Dravet Syndrome Foundation (DSF) is accepting applications for its Patient Assistance Grants (PAG) program that provides financial aid to people with Dravet syndrome who need medical equipment, therapy devices and educational tools. Since 2009, the PAG program runs from March 1 to Dec. 1 or until the funds…

Takeda Buys Global Rights to Soticlestat for Rare Epileptic Disorders

Takeda Pharmaceuticals has acquired the global rights to develop and market the investigational oral therapy soticlestat (OV935/TAK-935) for developmental and epileptic encephalopathies, including Dravet syndrome, the company has announced. Soticlestat was originally developed by Takeda and Ovid Therapeutics, after the companies entered into a collaboration in 2017.