efficacy of stiripentol fenfluramine and their combination on clinical outcomes in dravet syndrome a preliminary report

Original title: “Efficacy of stiripentol, fenfluramine, and their combination on clinical outcomes in Dravet syndrome: A preliminary report.”

from first seizure to specific antiseizure medication in dravet syndrome quantifying delays in the ds covered study

Original title: “From first seizure to specific antiseizure medication in Dravet syndrome: Quantifying delays in the DS'coverED study.”

practical consensus recommendations for polytherapy involving stiripentol in dravet syndrome a nominal group approach

Original title: “Practical consensus recommendations for polytherapy involving stiripentol in Dravet syndrome: A nominal group approach.”

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and well tolerated by patients with Dravet syndrome for whom antiseizure medications are not effective. The most common TEAE in the Phase 1/2a trials was post-lumbar puncture syndrome (nearly 25%), while the most common event in the extension studies was cerebrospinal fluid (CSF) protein increase (45%). However, none of the patients with CSF protein increase had increased intracranial pressure or hydrocephalus. Of the serious TEAE, only one was considered treatment-related. "Our data support zorevunersen safety and tolerability, as well as improvement in overall clinical status, quality of life, and adaptive behavior following continued dosing in the extension studies," said Dr. Owen – a Lurie Children's patient who participated in the clinical trial and now continues in the open-label extension study – is a 12-year-old boy with Dravet syndrome, whose seizures were not controlled by medications. He also had intellectual disability and gait abnormalities. Brain microphysiological systems are reshaping in vitro neurotoxicity testing through functional validation and advanced disease modeling. Results, published in the New England Journal of Medicine, include significant seizure reduction and improvement in other symptoms of Dravet syndrome, such as language, motor, and behavior issues. Researchers also report sustained treatment benefits in ongoing open-label extension studies.

Original title: “Novel gene regulation therapy passes key clinical milestone”

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial results. The drug, called zorevunersen, holds promise, especially for people with the condition who don’t respond to existing treatments such as antiseizure medications. New gene therapy results bring hope for treating Dravet syndrome, a rare and often fatal seizure condition Over the course of 20 months, patients had between 59 percent and 91 percent fewer seizures than before they started the treatment. Most side effects were moderate or mild. The studies were designed to test the safety and tolerability of the medication at varying dosages. Another study, a phase 3 randomized control trial, is underway, and it will more rigorously test how well the medication treats Dravet syndrome’s core symptoms. But the data from the studies published today suggest that zorevunersen is treating the underlying cause of the disease. Doctors currently prescribe antiseizure drugs and therapeutic diets to manage the seizures that characterize the condition. But these treatments are often ineffective. “It’s very rare that [a patient becomes] seizure-free,” says senior author Helen Cross of University College London. “These results are incredibly promising, and the levels of improvements are unprecedented in this disease state,” says Veronica Hood, chief science officer of the Dravet Syndrome Foundation. “This level of improvement is meaningful in so many aspects of daily life.” · Eight-year-old Freddie Truelove of Huddersfield, England, has Dravet syndrome and has taken part in a trial of zorevunersen, an ASO gene therapy to treat the condition.

Original title: “Pioneering gene therapy may treat a deadly seizure disorder | Scientific American”