A $25 million gift will be given to Penn Medicine and the Philadelphia Children’s Hospital. the Center for Epilepsy and Neurodevelopmental Disorders’s founding principles

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Children’s Hospital of Philadelphia and Penn Medicine Receive $25 Million Donation to Establish Center for Epilepsy and Neurodevelopmental Disorders

Philadelphia Children’s Hospital and Penn Medicine to Receive His $25 Million Donation to Establish the Center for Epilepsy and Neurodevelopmental Disorders

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The Center for Epilepsy and Neurodevelopmental Disorders (ENDD) donated her 2,500 from unnamed donors to Pen Medicine and Children’s Hospital of Philadelphia (CHOP) to conduct collaborative research on gene therapy to advance neurodevelopmental disorders. Founded thanks to a $1 million grant. Your donation will enable Penn and CHOP’s interdisciplinary team of doctors and researchers to advance their work. When it turned out, Dr. Prosser, an associate professor of physiology at the University of Pennsylvania’s Perelman School of Medicine, pointed the direction of his research career. J. Larry Jameson, MD, PhD, Vice President for Health Systems at the University of Pennsylvania and Dean of the Perelman School of Medicine, said ENDD: “This important contribution accelerates the development of an amazing collaboration between PEN and CHOP that demonstrates the value of teamwork and commitment to treating disease. I am very grateful to this contributor for working on it, which will undoubtedly open the way to new perspectives for those living with these conditions.

This donation was made by his son Stephen B. Burke served on the board of CHOP.

Madeline Bell, president and CEO of Children’s Hospital of Philadelphia, said: “We support our efforts to treat incurable patients with rare diseases and bring new knowledge about potential treatments to the clinic. We are deeply grateful for this inspiring gift that strengthens our ability to put it into practice. This new facility will bridge the tremendous gap between developing new treatments for these patients and making significant progress in early detection and intensive clinical care. .

The STXBP1 and SYNGAP1 genes are associated with abnormal brain function, intellectual disability, epilepsy, movement and behavioral disorders. ENDD intends to initially focus on developing treatments for these disorders and eventually to extend its efforts to other hereditary neurodevelopmental disorders. ENDD is supported by Drs. Prosser and Dr. Davidson, Director and Chief Scientific Strategy Officer of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at CHOP and Professor of Pathology and Laboratory Medicine at the University of Pennsylvania, and Drs. Dr. Helbig is a pediatric neurologist in the University of Pennsylvania Department of Neurology and CHOP Neurology, and the Genomics Director of CHOP’s Epilepsy Neurogenetics Initiative (ENGIN). Collaborative efforts at the center will focus on developing antisense oligonucleotides (ASOs), gene therapy, and CRISPR-based NDD therapeutics.

The daughter of Dr. Lucy Prosser. Prosser has been identified as a rare genetic neurodevelopmental disorder caused by mutations in the STXBP1 gene. Seizures, developmental delay, intellectual disability, and movement disorders are just a few of the symptoms that STXBP1-related disorders can cause. There are no known treatment options or cures.

Dr. Prosser, an expert in cardiac molecular biology, is focused on developing new drugs for STXBP1 and related diseases out of a desire to help his daughter and his more than 5,000 children, who are born each year around the world. He also started his own research field in his lab. Born with the STXBP1 problem. The ENDD Therapeutics Team was formed as a result of collaboration with Davidson, Helbig and other Penn and CHOP academics and medical professionals.

Prosser, as a researcher and a father, thanked the team for the gift of advancing their research in hopes of changing these circumstances. You have a unique opportunity with an amazing and dedicated team of PEN and CHOP scientists and physicians who are motivated every day to bring about

As a pioneer in gene therapy for neurological diseases, Davidson investigates the molecular basis and development of genetic and small-molecule therapeutics for pediatric neurodegenerative diseases. He Helbig of CHOP, one of the leaders of the Epilepsy Neurogenetics Initiative (ENGIN), specializes in SYNGAP1 and STXBP1 and has treated many children with these disorders, including Lucy Prosser. The development of new therapies for neurodevelopmental disorders is being accelerated by an extraordinary blend of clinical expertise, gene therapy innovations, and the enthusiasm of parents and scientists.