Our Focus: Genetically Defined Neurological Disorders

Scientific breakthroughs in our understanding of the genetic underpinning of many diseases have ushered in a new era of drug discovery reminiscent of the advances that have revolutionized cancer treatment over the last several decades. From epilepsy to neuropsychiatric disorders, these insights are driving the discovery of new therapeutic targets and precision medicines with the potential to significantly improve treatment for a broad range of patients.

Rare Epilepsy

Epilepsy genomics have created a new frontier for rational therapies. Through initiatives such as epi4K, Epilepsy Phenome/Genome Project, Epilepsy Genetics Initiative and EpiPM Consortium, we are rapidly developing a new understanding of the genetic drivers of epilepsy, including uncovering novel biological targets. With these targets in hand, we finally have a roadmap for developing precision medicines that have the potential to transform the lives of families affected by rare, debilitating epilepsies.

Psychiatric and Movement Disorders

Genomic research in epilepsy is providing important new insights into disorders of neuronal signaling, providing opportunities to treat challenging psychiatric and movement disorders by targeting previously unappreciated biology. Research has demonstrated that epilepsy pathways converge with those of other neurological conditions, such as bipolar disorder, ataxias, pain, autism and schizophrenia, allowing us the potential opportunity to explore investigational therapies in a broader range of diseases.

To inquire about participation in our clinical trials, please contact [email protected]