For families living with Dravet syndrome, 2025 has been a year filled with moments of hope, progress, and connection. Our community has come together to celebrate not only scientific breakthroughs but also the bonds that make this difficult medical journey a little less daunting.
Breakthroughs on the Horizon
Imagine a child living with daily seizures, whose every day is measured in careful seizure logs, other medical complexities, and setbacks. For these families, news about zorevunersen, an anti-sense oligonucleotide (ASO) therapy developed by Stoke Therapeutics has been exciting. Early data from the Phase 1/2 study showed meaningful reductions in seizures, along with improvements in cognition and behavior. For many families, this is more than a new treatment – it represents the first real glimpse of a therapy that could modify the course of Dravet syndrome and offer a better quality of life for their loved one. And now, dosing in the Phase 3 trial is underway.
The momentum doesn’t stop there. ETX101, an AAV-based gene therapy from Encoded Therapeutics. entered its Phase 1/2 trial in June 2024. Families are watching closely, hopeful that each milestone brings them closer to a world where Dravet syndrome is no longer an unchangeable part of life, but a treatable condition. Each step forward in research is a step toward hope, toward possibility, and toward a future where children can live with fewer limitations and more opportunities to thrive.
Coming Together as a Community
In 2025, Dravet Syndrome Foundation (DSF) hosted five Day of Dravet workshops across the U.S., bringing together more than 500 community members, representing 123 patient families. These gatherings provided a unique space for parents to share their experiences, for siblings to find camaraderie, and for everyone to discover a support system that truly understands the challenges and triumphs of living with Dravet syndrome. In addition, UCB partnered with DSF to offer Camp Small Steps at each workshop. These mini-camp experiences for patient families took place in the evening and offered a variety of fun camp experiences – ranging from crafts to games to songs by the campfire. These events fostered lasting connections, strengthened the community, and reminded families that they are never alone on this journey.
In February, members of the Dravet community also took their voices to Capitol Hill during Rare Disease Week, advocating for policies and resources to support families like theirs. It’s a reminder that strength doesn’t just come from science – it also comes from standing together.
Investing in the Future
Behind the scenes, DSF continues to power discovery. In 2025 alone, more than $3 million will be invested by DSF to advancing research, addressing critical gaps in knowledge, and developing innovative approaches to treating Dravet syndrome. This brings the total funding awarded by DSF to over $14 million, reflecting a sustained commitment to supporting groundbreaking science that moves the field forward and brings hope to families affected by this condition.
On December 4th, our annual DSF Research Roundtable will convene leading researchers and clinicians from around the world to set the course for the next generation of breakthroughs in Dravet syndrome. This annual gathering not only sustains the momentum of scientific innovation but also strengthens the hope of families everywhere. Each dollar invested, every study launched, and every conversation among researchers brings the community one step closer to a future where Dravet syndrome is better understood, more effectively treated, and, ultimately, more manageable – transforming the lives of patients and their families.
Strengthening the Foundation
2025 marked the 16th anniversary of DSF – a milestone that reflects years of dedication, resilience, and unwavering commitment to families affected by Dravet syndrome. In this landmark year, DSF is also strengthening its infrastructure to better serve the community.
Our newly formed Patient Equity & Inclusion Committee plays a vital role in ensuring that every voice within our community is heard. By elevating diverse perspectives and addressing barriers to access, the committee helps guide our programs and initiatives so they reflect the needs and experiences of all families affected by Dravet syndrome.
In June, we hosted our State of the Foundation webinar, where we provided an in-depth update on our progress, shared key accomplishments, and outlined the strategic priorities guiding our work moving forward. The session offered families, partners, and supporters a clear view of where we stand today and where we’re headed as we continue advancing our mission.
We also expanded the expertise of our professional advisory boards with the addition of two new members. Adult neurologist Fábio Nascimento, MD, joined our Medical Advisory Board, bringing valuable clinical insight into the adult experience of Dravet syndrome. In addition, Northwestern University researcher Gemma Carvill, PhD, joined our Scientific Advisory Board, further enhancing our capacity to guide research priorities and scientific strategy.
Supporting Families When They Need It Most
For families living with Dravet syndrome and facing life’s most challenging moments, DSF is there every step of the way, providing both practical and emotional support. Patient assistance and disaster-relief grants help ease the financial and logistical burdens of care, ensuring that no family has to navigate these challenges alone. Through moderated support groups and monthly “Chat & Connect” sessions, families have a safe, welcoming space to share experiences, find understanding, and receive compassion from those who truly understand their journey.
In partnership with the Mason’s Movement Foundation, families grieving the loss of a loved one find comfort, guidance, and meaningful connection during some of life’s most difficult moments. Building on this foundation, in 2025 DSF launched the Bereavement Grant program, offering additional resources to support families as they navigate loss – providing care, guidance, and compassion when it is needed most.
2025 has been a Year of Gratitude and Progress
For the Dravet community, it’s a reminder that every breakthrough, every connection, and every act of support brings families closer to a future filled with possibility. In a world where uncertainty is a constant companion, this year has given families something to celebrate: the power of progress and the strength of community.
