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