NAIROBI, Kenya — A new experimental therapy is offering fresh hope to children born with a severe form of epilepsy known as Dravet syndrome, after early research showed the treatment could dramatically reduce seizures by targeting the genetic cause of the disorder.
The drug, known as Zorevunersen, is designed to address the underlying biological mechanism that triggers the condition rather than simply controlling symptoms.
Scientists say the approach represents a significant step toward precision medicine for rare neurological diseases.
According to findings published in the New England Journal of Medicine, early clinical trials have shown promising results, with researchers reporting that the therapy can be safely administered to children as young as two years old.
Dravet syndrome is a rare but severe epilepsy disorder that typically begins during infancy.
The condition often emerges within the first year of life and can lead to frequent and prolonged seizures.
The World Health Organization estimates the disorder affects roughly one in every 15,000 births worldwide.
Children with the condition may experience dozens of seizures daily, placing them at constant risk of injury, developmental delays, and life-threatening complications if the seizures are not adequately controlled.
For many families, managing the condition remains extremely difficult.
Seizures can occur suddenly and may be triggered by fever, infections, or even minor changes in body temperature.
In severe cases, episodes can last longer than usual and require emergency medical care.
Unlike conventional anti-seizure medicines, the experimental drug aims to correct the genetic defect responsible for the disorder.
Most cases of Dravet syndrome are linked to mutations in the SCN1A gene.
This gene plays a critical role in how brain cells communicate. It produces sodium channels that allow electrical signals to pass between neurons.
When the gene malfunctions, the brain produces fewer of these channels, disrupting communication between cells and triggering abnormal electrical activity that leads to seizures.
Researchers say the therapy works by boosting the activity of the healthy copy of the gene. This helps the brain produce more sodium channels and restore normal neural communication.
The treatment is delivered through an infusion into the lower back, allowing the drug to travel through spinal fluid directly to the brain, where it is needed most.
Early trial results suggest the therapy could significantly reduce seizure frequency. Researchers reported that some children receiving repeated doses experienced seizure reductions of up to 90 per cent.
The development comes amid growing global concern about the burden of epilepsy. Worldwide, an estimated 50 million people live with the condition, making it one of the most common neurological disorders.
However, nearly one in three epilepsy patients continues to experience seizures that do not respond well to existing medications.
In Kenya, epilepsy also affects thousands of families.
Studies suggest that between 11 and 41 out of every 1,000 children may be living with some form of the condition, highlighting persistent gaps in diagnosis, treatment, and specialist neurological care.
Although Dravet syndrome represents only a small fraction of epilepsy cases, experts say the condition is frequently underdiagnosed, particularly in low- and middle-income countries where access to genetic testing remains limited.
The disorder often begins when a baby is between four and eight months old.
The first seizure commonly occurs during a fever or illness, even in children who had previously appeared healthy.
Beyond seizures, the syndrome can affect a child’s overall development.
Many patients experience speech and learning delays, problems with balance and coordination, and behavioural challenges that complicate daily life for both children and their caregivers.
Currently, there is no cure for Dravet syndrome. Treatment typically focuses on reducing seizure frequency and improving quality of life through combinations of anti-seizure medications.
Some families also turn to additional therapies such as the ketogenic diet, which can help reduce seizures in certain cases.
In other situations, doctors may recommend devices like vagus nerve stimulators to help control brain activity.
Scientists say emerging therapies that directly target the genetic causes of neurological disorders could transform how rare diseases are treated.
While Zorevunersen is still under investigation and requires further clinical trials before regulatory approval, researchers say the early results represent an encouraging step toward more effective treatments for children living with Dravet syndrome.
