Therapeutic Focus

What is epilepsy?

Epilepsy is a serious and chronic neurological disorder characterized by recurrent and unprovoked seizures caused by abnormal brain activity. Recurrent seizures can not only severely disrupt patients’ quality of life, but epilepsy patients also experience higher mortality risk compared to the general population. Epilepsy is one of the most prevalent neurological disorders and affects an estimated 50 million individuals globally. It affects people of all ages, genders, and races.

Limitations of current medications

While there are multiple anti-epileptic drugs available to patients, most epilepsy patients still experience significant unmet needs.

PillDrug resistance – One third of patients are drug-resistant, failing to respond to two or more anti-epileptic drugs
PillLimited efficacy – Many patients see limited benefit from one or multiple anti-epileptic drugs, which is exacerbated by the many presentations of epilepsy
PatientSide effects – The risk of side effects from anti-epileptic drugs is harmful. This risk is heightened when multiple drugs are used in combination

2000 patientsDravet syndrome is a rare and drug-resistant form of epilepsy that begins in infancy and significantly impacts patients throughout their lifetime. There are an estimated 20,000 Dravet syndrome patients in the US and EU. Approximately 80% of Dravet patients have a SCN1A gene mutation that disrupts the sodium ion channels and hence electrical signals in the brain.

100,000 patientsLennox-Gaustat syndrome is a rare and drug-resistant form of epilepsy that begins around 3-5 years of age and significantly impacts patients throughout their lifetime. There are an estimated 100,000 LGS patients in the US and EU. The causes of LGS are wide-ranging, with genetic differences and structural differences of the brain being the two major causes.

20 percentRefractory, genetically related epilepsies account for approximately 20% of epilepsies or 1 million patients in the US and EU. Epilepsies are considered genetically-related if seizures are the result of a known or presumed genetic problem. Genetically-related epilepsies can present with a broad range of seizure types.

Dravet syndrome

Dravet syndrome is a rare and drug-resistant form of epilepsy that begins in infancy and significantly impacts patients throughout their lifetime. There are an estimated 20,000 Dravet syndrome patients in the US and EU. Approximately 80% of Dravet patients have a SCN1A gene mutation that disrupts the sodium ion channels and hence electrical signals in the brain.

Lennox-Gastaut Syndrome

Lennox-Gaustat syndrome is a rare and drug-resistant form of epilepsy that begins around 3-5 years of age and significantly impacts patients throughout their lifetime. There are an estimated 100,000 LGS patients in the US and EU. The causes of LGS are wide-ranging, with genetic differences and structural differences of the brain being the two major causes.

Refractory, genetically-related epilepsies

20 percent
Refractory, genetically related epilepsies account for approximately 20% of epilepsies or 1 million patients in the US and EU. Epilepsies are considered genetically-related if seizures are the result of a known or presumed genetic problem. Genetically-related epilepsies can present with a broad range of seizure types.

Resources for patients and families

dravet syndrome foundation
american epilepsy society
dravet canada
nord
sindrome de dravet
epilepsy foundation
dravet syndrome uk
lgs foundation

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