Published: Sept 2025
Dravet Syndrome UK attended the 35th International Epilepsy Congress (IEC) in Lisbon, where we not only attended key presentations but also connected with leading clinicians, pharmaceutical developers focused on new treatments for Dravet Syndrome, and other patient organisations. Our Chief Scientific Officer, Ceri, has highlighted the key themes that emerged from this conference.
IEC 2025 reinforced a shared commitment among patients, clinicians, researchers, and advocates alike. Together, this collaborative community is driving meaningful progress toward better outcomes and improved quality of life for everyone impacted by Dravet Syndrome.
Empowering Patients
The Presidential Symposium, titled “Patient-centred healthcare: from challenge to change,” set the tone for IEC 2025 by amplifying patient voices in research and care discussions. In many sessions, patient representatives, including DSUK’s Chair or Trustees, Galia Wilson, shared their stories. Galia shared the benefit of receiving a genetic diagnosis that enabled her son to access better treatments and helped her family find a supportive community.
Patient advocates highlighted the benefits of patient and public involvement (PPI); the The Mental Health Intervention for Children with Epilepsy (MICE) Project study team presented evidence showing that PPI offers patients a sense of purpose and provides researchers with deeper insights into what truly matters to the community. The first-time presentation of the IBE’s Global Epilepsy Needs Study revealed patient priorities such as seizure unpredictability, difficulties accessing healthcare, and the urgent need for specialised complex epilepsy services. This large-scale study is a vital educational tool for healthcare professionals, policymakers, and researchers in recognising patients’ holistic needs.
IEC 2025 also delivered important messages supporting transparent conversations with patients and families about SUDEP, status epilepticus, nutritional issues, and long-term decline. Providing this knowledge early ensures that preventative strategies can be put in place, as patients cannot address risks they do not know about.
Collaboration, communication, and education remain essential to empower patients as active partners in shaping research and care.
Rethinking Clinical Trial Design
Discussions reflected that the era of placebo arms, especially in invasive or high-risk studies, is evolving, with natural history studies offering a compelling and more ethical alternative. Presenters acknowledged the ongoing SCN1A Horizons Natural History study and its potential to provide baseline progression data for people with Dravet Syndrome; this could eliminate the need for a control cohort and provide more equitable access to treatment during trials.
Furthermore, there was recognition that measuring trial success solely by a reduction in motor seizures does not reflect the broader goals families and clinicians share. Participants highlighted the importance of capturing changes in seizure length and severity, as well as cognitive and behavioural improvement. Identifying and developing appropriate tools to measure changes in these domain remains a hurdle in making these endpoints commonplace. The addition of biomarkers to outcome measures was discussed not only to bring objective clinical data but also as a method to include people with non-motor seizure types.
Finding the right balance between regulatory requirements and clinically meaningful changes will be critical for making future therapies more accessible and valuable for patients and families.
Disease Modification updates
The congress featured passionate calls from experts like Ana Mingorance to continue advancing disease-modifying treatments for Dravet Syndrome and related developmental and epileptic encephalopathies. Two particularly promising advances for Dravet Syndrome received attention at IEC 2025.
Encoded Therapeutics’ ETX101 gene therapy trial for Dravet Syndrome has received the U.S. FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation. While not yet influencing UK regulatory pathways directly, this reflects encouraging early data. ETX101 aims to upregulate the healthy copy of SCN1A and, if successful, could offer a one-time treatment with lasting impact.
Stoke Therapeutics presented three-year open-label extension results for zorevunersen, a disease-modifying antisense oligonucleotide treatment. Long-term data show substantial, durable reductions in convulsive seizures, alongside clear improvements in cognition and behaviour. If phase 3 studies confirm these benefits, zorevunersen has potential have a lasting impact on patient quality of life.
These advances reinforce hope for meaningful disease modification, moving beyond seizure control and towards truly altering the course of Dravet Syndrome.