Patricia Inácio, PhD, science writer —

BL-001, an investigational oral therapy for Dravet syndrome, was well-tolerated across multiple-ascending doses in healthy volunteers, top-line data from a Phase 1 trial show. These results indicate that BL-001 can be dosed at concentrations up to 10-fold higher than the most effective dose in animal models, according to…

Baylor Genetics is offering a gene analysis service on epileptic disorders, including conditions like Dravet syndrome. The specialized panel is designed for outpatient use and is intended to assist clinicians in assessing the genetic factors underlying a patient’s epilepsy. “When a genetic cause of epilepsy is suspected, a…

Diacomit (stiripentol) was safe and clinically effective at reducing seizures when given to infants with Dravet syndrome, ages 2 or younger, according to a 30-year real world study. It also reduced emergency hospitalizations due to long-lasting seizures. “This 30-year, real-world experience shows that initiating stiripentol before the age of…

Children and adults with Dravet syndrome commonly exhibit challenging behaviors such as aggression, dangerous behavior, and impulsivity, according to a Dutch study based on responses from 20 parents. These behaviors, apart from the seizures that are a hallmark of the disease, can greatly affect the quality of life of…

A Phase 1 clinical trial that enrolled healthy volunteers to test BMB-101, Bright Minds Biosciences’ investigational treatment for Dravet syndrome, has moved to its final part, the company announced in a corporate update. This first-in-human trial of the oral therapy, which is testing BMB-101 at different doses,…

The efficacy and safety of Epidiolex, an oral cannabidiol, in children and adults with Dravet syndrome and Lennox-Gastaut syndrome (LGS) were supported in a real-world setting, with benefits similar to those seen in previous clinical trials and expanded access programs (EAPs), a Spanish EAP study reported. EAPs are…

STK-001, Stoke Therapeutics’ investigational therapy, continues to show promise in safely reducing seizures among children and adolescents with Dravet syndrome, according to new interim data from the ongoing Phase 1/2a MONARCH and ADMIRAL trials. “We are encouraged by the data from our ongoing studies of STK-001, which continue…

Children and adolescents with Dravet syndrome with walking difficulties commonly experience impairments in functional mobility and greater restrictions in daily life activities, a study suggests. Its researchers recommend that physiotherapy be given patients to improve their gait, potentially allowing for better mobility in daily life. The study, “…

Note: This story was updated Feb. 7, 2022, to clarify that CAMP4 will continue preclinical studies for its lead candidate. Also, CAMP4 licensed the antisense oligonucleotides from OPKO Health, not the RNA Actuating Platform, which was developed in-house. CAMP4 Therapeutics‘ investigational RNA-based therapy — called CMP-SCN (CO-3527) — increased…

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.