Division of Neurology News

The award will fund the Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform – a collaboration of academic researchers, clinicians, patient advocates, and biotechnology companies aiming to develop gene-editing treatments for children with rare forms of epilepsy.

Using adenine base editing, the team efficiently corrected the disease-causing DNA change in a preclinical in vivo model.

New FDA-approved therapy significantly reduces pain associated with migraine disease in pediatric patients

Children’s Hospital of Philadelphia (CHOP) today announced the establishment of the Elise’s Corner Endowed Chair in Alexander Disease Research. Dr. Amy T. Waldman, Medical Director of the Leukodystrophy Center and a pediatric neurologist at CHOP, has been named the inaugural chair holder. Funded by Elise’s Corner and Jim and Marguerite Pearson, the role will support research and treatments for Alexander disease, a form of leukodystrophy that progressively damages the nervous system.

Children’s Hospital of Philadelphia (CHOP) is proud to announce the selection of Christina Gurnett, MD, PhD, a nationally recognized expert in pediatric and developmental neurology, as its new Chief of the Division of Neurology and Co-Director of the Neuroscience Center. Gurnett began her new role at CHOP on September 1, 2025.

Researchers identified a novel gene associated with neurodevelopmental disorders and epilepsy. The study leveraged large data depositories, state-of-the-art computational techniques and community-based gene matching to identify this new gene.

Researchers at Children’s Hospital of Philadelphia (CHOP) discovered a previously unrecognized brain event following electroconvulsive therapy (ECT), the most clinically effective treatment for severe, medication-resistant depression. The findings, which could provide additional insight into the biological mechanism of ECT, were recently reported in the journal Nature Communications.

Researchers have found that in rare instances, variants responsible for SYNGAP1-related disorders – a group of disorders characterized by developmental delay and often associated with epilepsy – can be inherited from a parent, which could help influence family planning, genetic variant interpretation, and other aspects of clinical care.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania announced the first ever direct approach to the depletion and replacement of microglia, the native immune cells of the central nervous system (CNS).

Researchers discovered increased incidence of specific seizures both in infancy and later in childhood that could help explain disease trajectory and set important clinical trial targets