Researchers at Children’s Hospital of Philadelphia (CHOP) developed a longitudinal atlas of neuroblastoma, a common and potentially deadly childhood cancer, to gain deeper understanding into precise molecular mechanisms underlying why and how certain treatments eventually become ineffective.

New research from Children’s Hospital of Philadelphia (CHOP) offers insight into how a rare genetic condition called Beckwith–Wiedemann syndrome (BWS) affects the liver and may raise cancer risk.

To reduce staff stress and burnout, researchers piloted a 12-session resilience program in two West Philly K-8 schools and evaluated its impact and adaptability.

In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.

New research from Children’s Hospital of Philadelphia (CHOP) uncovered a previously unknown side effect of Acute Myeloid leukemia (AML), the second most common blood cancer in children, that leaves a long-lasting imprint on healthy blood-forming stem cells, influencing immune responses long after the disease has entered remission. The results were published recently in the journal Cancer Letters.

Cardiology 2025, the 28th Annual Update on Pediatric and Congenital Cardiovascular Disease, was held this year from February 19 to 23 at Disney’s Yacht & Beach Club Report in Lake Buena Vista, Florida. The conference, hosted by Children’s Hospital of Philadelphia (CHOP), brought together a dynamic and diverse community of pediatric and congenital cardiovascular professionals and advocates, all united by this year’s theme: Hope, Heal and Learn.

Children’s Hospital of Philadelphia (CHOP) is proud to announce awards from the Eagles Autism Foundation totaling more than $1.3 million over the course of the next two years. The awards will support five cutting edge autism projects from various research labs across CHOP’s research enterprise.

Researchers in the Center for Violence Prevention (CVP) at Children’s Hospital of Philadelphia (CHOP) collaborated with a team led by Lurie Children’s Hospital in Chicago that found children living in areas with the lowest Child Opportunity Index (COI), a measure that assesses factors like education, socioeconomic resources and access to health environments, were more than twice as likely to experience another violent injury within a year of an initial firearm related emergency department (ED) visit. The retrospective study was published recently in the journal JAMA Pediatrics.

In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment for metachromatic leukodystrophy (MLD), a rare disease in young children characterized by the deficient activity of a critical enzyme. Without effective treatment, MLD leads to rapidly declining neurological skills in young children and is potentially fatal. The promising findings were published in the journal Molecular Therapy Nucleic Acids.

On Saturday, February 22, 2025, Children's Hospital of Philadelphia (CHOP) hosted Cheers for CHOP, presented by Calvin Schmidt and family, at The Fillmore. The event benefitted rare disease research at CHOP, where rare cases are not so uncommon. Every dollar of the $745,000 (and counting!) raised will help our teams continue their work of translating rare disease research into more precise and personalized care for patients with some of the most complex medical cases.