Penn Medicine and CHOP: $25 Million Donation to Establish an Epilepsy and Neurodevelopmental Disorders Center
A $25 million donation from an anonymous donor to Penn Medicine and Children’s Hospital (CHOP) in Philadelphia establishes the Center for Epilepsy and Neurodevelopmental Disorders (ENDD) to accelerate research collaborations in gene therapy for neurodevelopmental disorders increase. The donation strengthens the efforts of a multidisciplinary group of PEN and CHOP clinicians and scientists, led by director Benjamin Prosser and co-directors Beverly Davidson and Ingo Helbig. I am an Associate Professor of Physiology at the University of Medicine. He adjusted the focus of his research career when his daughter Lucy was diagnosed with a rare hereditary neurodevelopmental disorder shortly after her birth in 2018.
“ENDD brings hope to patients and families living with the many challenges of a rare inherited neurodevelopmental disorder,” said J. Larry Jameson says. “This important gift accelerates the progress of the extraordinary partnership between PEN and CHOP, demonstrating the power of collaboration and our commitment to finding cures. We are deeply grateful to this donor whose dedication will ensure it creates a starting point of new perspectives for those diagnosed with these diseases.”
This gift was made in honor of the late television executive Daniel B. Burke, whose son Stephen B. Burke was a member of the CHOP Board of Trustees.
Madeline Bell, President and CEO, Children’s Hospital of Children’s Hospital, said: Philadelphia. “CHOP and Penn have systematically invested in integrative treatment programs for hereditary epilepsy and neurodevelopmental disorders. It will bridge the gap between the development of new therapies for
ENDD initially focused on developing therapeutics for disorders associated with mutations in the STXBP1 and SYNGAP1 genes associated with abnormal brain function, intellectual disability, epilepsy, movement and behavioral disorders, and extended its efforts to other genetic neurological disorders. The goal is to extend to developmental disabilities. over time. ENDD, Dr. Prosser and Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, CHOP’s Chief Scientific Strategy Officer and Professor of Pathology and Clinical Medicine at the University of Pennsylvania, his Dr. Davidson and Dr. Strengthen existing partnership with Davidson. Dr. Helbig is a pediatric neurologist at the Department of Neurology at the University of Pennsylvania and he is the Director of Genomic Sciences for CHOP’s Epilepsy Neurogenetics Initiative (ENGIN). Their collaboration at the Center targets the development of antisense oligonucleotides (ASOs), gene therapy, and CRISPR-based strategies for the treatment of NDD.
Dr. Prosser’s daughter Lucy was diagnosed with a rare hereditary neurodevelopmental disorder caused by mutations in the STXBP1 gene. STXBP1-related disorders cause a variety of symptoms, including seizures, developmental delay, intellectual disability, and movement disorders. There is no known cure or effective cure.
Dr. Prosser, an expert in cardiac molecular biology, is motivated by his daughter and the desire to help the more than 5,000 children with STXBP1 disorders each year around the world who are interested in developing STXBP1 and related new treatments. He set up a separate research unit in his lab to focus on. hindrance. He started working with Dr. Davidson and Helbig, as well as other researchers and clinicians from Penn and CHOP, form his ENDD treatment team.
“As scientists and as parents, we are immensely grateful for this gift that advances our research in the hope of changing the course of these disorders,” Dr. Prosser said. , to have a rare opportunity to work with such a talented and dedicated team of pen and CHOP scientists and clinicians who are motivated every day to make a difference for Lucy and children like her. .”
A world-renowned expert in gene therapy in neurological disorders, Dr. Davidson is committed to understanding the molecular basis of childhood-onset neurodegenerative diseases and developing therapeutic gene and small-molecule therapies. increase. He is one of the directors of the Epilepsy Neurogenetics Initiative at CHOP. increase. This unique combination of clinical knowledge, gene therapy innovation, and parent scientist motivation will accelerate the development of new therapies for neurodevelopmental disorders.
