Epilepsy: Developing Personalized Therapies

Our Breakthrough

Developing Personalized Epilepsy Therapies

Individualized, gene-specific treatment plans for patients

Our Challenge

Epilepsies That Are Difficult to Treat

Epilepsy is one of the most common chronic neurological conditions of childhood, affecting 1 to 2 percent of all children in the United States; more than half of these cases have a genetic basis. Many epilepsies are unexplained and difficult to treat.

Our Solution

Precision Therapies

Develop precision therapies for genetic epilepsies using experimental model systems, including neurons and organoids (“minibrains”) generated from patient-derived induced pluripotent stem cells.

Our Impact

More Options for Children

We are creating individualized, gene-specific treatment plans that include medication, dietary treatment and epilepsy surgery. These treatments are life-changing for children.

Meet Ryan

Our Team

Leading the Way

Our team of neurogenetics experts works together to integrate genetics into the diagnosis and care of children with epilepsy – and to develop individualized treatment plans for each patient.

Ethan Goldberg, MD, PhD

Director, Epilepsy Neurogenetics Initiative

Katherine L. Helbig, MS, LCGC

Co-Director, Epilepsy Neurogenetics Initiative

Ingo Helbig, MD

Attending Physician, Division of Neurology

Xilma Ortiz-Gonzalez, MD, PhD

Attending Neurologist, Division of Neurology

Moving Ahead

Using “Big Data” to Improve Care

ENGIN researchers are analyzing data from patients’ electronic medical records and using it to pinpoint genetic mutations, identify optimal treatments and predict complications.

Find out how they used big data to identify a new gene associated with severe childhood epilepsy.

ENGIN investigators have been involved in the identification of more than 30 genetic epilepsy syndromes.

Help Us Find Solutions

We find real-world, scalable solutions to seemingly intractable problems. Led by the greatest minds in medicine, and with support from our generous donors, our world-renowned teams are taking on the toughest diseases the world has ever seen – and winning.