For families living with Dravet syndrome, progress can feel painfully slow. Seizures persist, comorbidities evolve, and the day-to-day realities remain complex. When you are in the middle of it, it’s hard to see change.
And yet, when we take a step back, the progress in Dravet syndrome over the past two decades is truly extraordinary. My son Elliot, now 26, is living in a time where disease-modifying therapies – something I never dreamed would be possible in his lifetime – are now actively in clinical trials.
From Unknown to Understood
Not long ago, Dravet syndrome was poorly understood and often misdiagnosed. It wasn’t until 2001 that researchers identified mutations in the SCN1A gene as a primary cause.
That discovery fundamentally changed the trajectory of the field. Today, more than 95% of diagnoses are linked to genetic findings, enabling earlier and more accurate diagnosis, and helping clinicians avoid treatments that may worsen seizures.
The shift from symptom-based recognition to genetic understanding laid the foundation for everything that followed.
From No Options to Targeted Therapies
Families who have been in this community for years remember a time when there were no treatments specifically approved for Dravet syndrome. Care was largely trial-and-error.
Today, that landscape looks very different.
Research has led to therapies like stiripentol (Diacomit), cannabidiol (Epidiolex), and fenfluramine (Fintepla) – each demonstrating meaningful reductions in seizure frequency in clinical trials.
Even more encouraging, the field is now moving beyond symptom management toward disease-modifying approaches. Emerging therapies, including gene therapies and antisense oligonucleotides, are designed to target the underlying cause of Dravet syndrome, not just its symptoms.Recent early-stage trials of therapies like zorevunersen suggest we may be entering an era where we can actually change the course of this disease.
That is a profound shift.
From Isolation to a Global Research Ecosystem
In the early 2000s, research activity in Dravet syndrome was minimal, with fewer than 30 scientific publications per year. Today, there are hundreds annually, reflecting a vibrant and growing field of inquiry.
This growth did not happen by accident.
It was fueled by a coordinated effort among families, clinicians, researchers, industry, and advocacy organizations working together with urgency and purpose.
The Role of Dravet Syndrome Foundation
Since its founding in 2009, Dravet Syndrome Foundation (DSF) has served as a catalyst for this progress.
- DSF has directed more than $14.1 million into research
- This early stage funding model has helped DSF funded researchers secure more than $35 million in subsequent National Institutes of Health (NIH) for projects focused on Dravet syndrome
- We has supported 84 research projects, seeding new ideas and accelerating innovation
Beyond funding, DSF has played a critical role in building infrastructure for progress by working as a convenor in the space. We have brought together stakeholders, supported natural history studies, and ensured that the patient voice guides research priorities.
Over the past 16 years, the community, through DSF, has helped drive advancements in diagnosis, treatment, awareness, and collaboration.
This is what collective action looks like.
Why It Still Feels Slow
Despite all of this progress, the unmet need remains significant. Many individuals with Dravet syndrome continue to experience uncontrolled seizures and significant comorbidities. And when it’s your child or loved one who is impacted, science will never feel like it’s moving fast enough.
Scientific advancement is inherently incremental. Each discovery builds on the last. Clinical trials take time. Regulatory pathways are rigorous by design. For families, that pace can feel misaligned with urgency, and understandably so.
But the trajectory is clear: progress is accelerating.
Looking Ahead: The Power of Strategy
As we enter the next phase of this work, intentional strategy matters more than ever.
Our strong strategic plan allows us to:
- Prioritize the most promising scientific opportunities
- Invest in areas that will have the greatest impact on patients
- Strengthen collaboration
- Ensure that no effort is duplicated and no opportunity is missed
It transforms momentum into measurable progress.
The next breakthroughs in Dravet syndrome – whether in gene therapy, precision medicine, or improved standards of care – will not happen by chance. They will happen because of coordinated, strategic action.
A Community That Moves Science Forward
The story of progress in Dravet syndrome is not just a story of science – it is a community story.
It is families who refused to accept the status quo.
It is researchers who chose to focus their careers on Dravet syndrome.
It is clinicians, partners, and advocates working together, worldwide
And it is this foundation – built by our community – that continues to push the field forward.
Progress may not always feel fast.
But it is real.
It is meaningful.
And it is accelerating.
Our work is far from done, but we are closer than we have ever been.
