What is Dravet syndrome?
Dravet syndrome is a rare and severe form of epilepsy that typically begins within the first year of life. In most cases, it is caused by a change in a gene called SCN1A, which plays a key role in how brain cells regulate electrical activity.
Children with Dravet syndrome may experience:
- Frequent and difficult-to-control seizures
- Developmental delays
- Challenges with learning, movement or behavior
- Increased risk of serious epilepsy-related complications
Because the condition is often linked to a known genetic change, researchers are working to develop therapies that move beyond seizure control alone.
How this new therapy is different
Most current epilepsy treatments focus on managing symptoms – mainly seizures. This investigational therapy takes a different approach.
Rather than replacing or editing genes, the study medication is designed to increase the activity of the child’s existing, working copy of the SCN1A gene. It uses a targeted RNA-based strategy intended to help brain cells produce more of a critical protein involved in normal nerve signaling.
Researchers are studying whether this approach may help address aspects of the condition at a molecular level. While it is still under investigation, it represents an important direction in epilepsy research.
What participation in the study involves
Children enrolled in this study receive the investigational therapy through scheduled spinal (intrathecal) injections administered while they’re under sedation, in a supervised medical setting. Participants continue to be closely monitored to assess therapy safety, tolerability and early signals of clinical impact throughout the study period.
Clinical trials follow strict safety protocols and include regular visits, evaluations and ongoing support from a specialized epilepsy care team. Phoenix Children’s specialists work closely with families to explain each step of the research process and answer questions along the way.
Why this research matters to families
For families affected by Dravet syndrome, even small advances can make a meaningful difference. second floor main hospital. Participation in studies like this reflect Phoenix Children’s broader commitment to:
- Advancing precision-based medicine – developing treatments matched to your child’s specific genetic cause
- Exploring therapies tailored to known disease mechanisms
- Expanding future treatment possibilities for children with complex epilepsy
Each study contributes valuable knowledge that helps shape the future of care. While investigational therapies are not guaranteed to provide a benefit, every family who participates helps build evidence that may benefit children with Dravet syndrome for years to come.
Comprehensive care beyond clinical trials
Participation in research is just one part of caring for children with Dravet syndrome and other complex seizure disorders. Families benefit from coordinated, long-term care provided by experienced specialists.
Phoenix Children’s Epilepsy Program brings together pediatric neurologists, epilepsy specialists, genetic experts, nurses and supportive services to care for children with even the most challenging forms of epilepsy. Whether or not a family chooses to participate in research, our team provides coordinated care and support.
Learn More
If your child has epilepsy or Dravet syndrome, expert guidance and compassionate care can make a difference. Phoenix Children’s offers comprehensive evaluation, advanced treatment options and access to ongoing research – focused on supporting patients and families at every stage.
To learn more about our Epilepsy Program contact our care team at 602-933-5437. You can also learn more about Dravet syndrome, including answers to frequently asked questions about diagnosis, treatment and what to expect.
