A preliminary report evaluates the efficacy of stiripentol and fenfluramine, both alone and in combination, on clinical outcomes in patients with Dravet syndrome. The findings contribute to understanding treatment options for this severe epilepsy disorder.

Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a ... Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function in the SCN1A gene, and China’s Center for Drug Evaluation has granted zorevunersen Breakthrough Therapy Designation. CEO Ian F. Smith and Chief Patient Officer Jason Hoitt will present at 1 p.m. ET, with a live webcast and archived replay on Stoke's website. Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability. ... A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Source: Stoke Therapeutics, Inc. ... Antisense oligonucleotides are short, synthetic pieces of genetic material designed to stick to specific messenger RNA molecules inside cells and block or alter the production of a particular protein. For investors, they matter because they represent a targeted drug approach with potential to treat diseases that other medicines cannot, but they also carry development, safety and regulatory risks that can lead to large swings in a biotech company’s value.

Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a ... Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function in the SCN1A gene, and China’s Center for Drug Evaluation has granted zorevunersen Breakthrough Therapy Designation. –4-year data from the Phase 1/2a open-label extension (OLE) studies support the potential disease-modifying effects of zorevunersen– –Improvements in... Biogen Safe Harbor This news release contains forward-looking statements, including, among others, relating to: the potential clinical effects of zorevunersen; the potential for zorevunersen to change the course of Dravet syndrome and improve outcomes for patients; the expected timing of Phase 3 study results; the potential benefits, safety and efficacy of zorevunersen; potential regulatory discussions, submissions and approvals and the timing thereof; the treatment of the underlying causes of Dravet syndrome; the anticipated benefits, risks and potential of Biogen's collaboration arrangements with Stoke Therapeutics; the potential of Biogen's commercial business and pipeline programs, including zorevunersen; and risks and uncertainties associated with drug development and commercialization. Based on Stoke Therapeutics’ preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by Wu et al., Pediatrics, 2015. ... August 31, 2026 19:30 ET | Source: Biogen Inc. Canada’s Drug Agency Issues Final Positive Recommendation Supporting Public Reimbursement of LEQEMBI® (lecanemab) for Early Alzheimer’s Disease

A recent study quantifies delays in administering specific antiseizure medications for patients with Dravet syndrome, highlighting the need for timely intervention. The DS'coverED study provides insights into the treatment timeline from the first seizure to medication initiation.

Information regarding the state-of-the-field for disease-modifying therapeutic approaches. Certain gene therapies work similarly by taking advantage of viral vectors, which are modified viruses that have been engineered to safely carry genetic material into cells. Viruses are naturally very good at entering cells, making them useful delivery tools once their disease-causing components have been removed. One of the most common vectors used in brain related gene therapies is the adeno-associated virus (AAV). AAV vectors are attractive because they efficiently deliver genetic material to neurons and generally do not cause disease in humans. Different types of AAV vectors may also target different tissues or cell types more effectively. No disease-modifying therapy for Dravet syndrome has yet been approved, and many important questions remain regarding long-term safety, durability, accessibility, timing of treatment, and which patients may benefit the most from each type of therapy. Nonetheless, the progress being made today represents an important step toward a future where treatments for Dravet syndrome may go beyond symptom management and directly address the underlying biology of the disorder. DSF has worked diligently to fund basic research that lays the groundwork for the development of genetic therapies. Several years ago, a genetic therapy still seemed a distant possibility for Dravet syndrome. However, advances to our understanding of genetics, neuroscience, and medicine have allowed researchers to develop new approaches to gene-based interventions, bringing the possibility of disease-modifying therapies closer to reality for Dravet syndrome.