Marta Figueiredo, PhD, science writer —

The blood levels of Fintepla (fenfluramine) are mostly within the lower range among treated children and adults with Dravet syndrome and other epileptic conditions, a study has found. Still, these levels were enough to achieve good seizure control in most Dravet patients, further supporting the therapy’s potent effects,…

Treatment with low-dose aripiprazole — a newer class of antipsychotic medication — in combination with psychotherapy, and behavioral and occupational therapy, was found to lessen behavior, attention, and educational problems in a 6-year-old girl with Dravet syndrome, according to a case study. This case points out a potentially effective…

Olivetolic acid, a precursor of cannabidiol (CBD), resulted in protective effects against fever-induced seizures in a mouse model of Dravet syndrome, a study shows. These effects were comparable to those previously reported with CBD in this model, suggesting that olivetolic acid may have the potential to reduce seizures in…

Nearly one month of repeated dosing of BMB-101, Bright Minds Biosciences’ experimental oral therapy for Dravet syndrome, was found to be safe and well-tolerated in mice and dogs. These were the findings of toxicology studies that began in September and are intended to support regulatory clearance of…

Stoke Therapeutics’ investigational therapy STK-001 is generally safe and results in a trend toward a reduction in seizure frequency in children and adolescents with Dravet syndrome, according to interim data from the ongoing Phase 1/2a MONARCH trial. These promising early findings, concerning STK-001’s single doses up to 30…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…

A no-cost genetic screening program, sponsored by Stoke Therapeutics, helped speed up molecular diagnosis of SCN1A-related diseases, including Dravet syndrome, by four years in children suspected of having genetic epilepsy, a study shows. Notably, clinical data from most of the children with SCN1A-related disorders suggested a future clinical diagnosis…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

The investigational oral therapy Soticlestat (OV935/TAK-935), developed by Ovid Therapeutics and Takeda Pharmaceuticals, safely and effectively lowers seizure frequency in children with Dravet syndrome and Lennox‑Gastaut syndrome (LGS), according to top-line data from a Phase 2 clinical trial. “We are extremely encouraged by these results, which…

Epilepsy surgery may be ineffective, and even detrimental, in people with Dravet syndrome caused by mutations in the SCN1A gene, a small multicenter study suggests. While patients with other SCN1A-associated epilepsies benefited from this type of surgery, Dravet patients did not, even in the presence of a…