Research News from CHOP


The Cardiac Center at Children’s Hospital of Philadelphia Hosts the 28th Annual Update on Pediatric and Congenital Cardiovascular Disease
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 Researchers Awarded $1.3 Million from Eagles Autism Foundation
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.
Pediatric Hospital Study Links Structural and Social Drivers of Health to Increased Recurrent Violence in Children with Firearm Injuries
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.

Children’s Hospital of Philadelphia Raises More Than $745,000 for Rare Disease Research at Cheers for CHOP
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.
Children’s Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy
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.
Children’s Hospital of Philadelphia Researchers Find New Protocol to Help Stabilize Youth Boarding in Emergency Rooms
Children’s Hospital of Philadelphia (CHOP) researchers successfully reduced the number of youths needing to be admitted to psychiatric facilities through a stabilization protocol designed to address psychiatric boarding, the practice of maintaining patients with psychiatric emergencies in the Emergency Department (ED) or other temporary settings due to a lack of available inpatient psychiatric beds. The report was published recently in the journal Pediatrics.
Children's Hospital of Philadelphia Researchers Identify Promising Interventions and Areas of Improvement for Addressing Microaggressions in Clinical Care
Microaggressions – brief and commonplace interactions that intentionally or unintentionally communicate insults and derogatory sentiments towards people of marginalized backgrounds – can happen anywhere.
Children’s Hospital of Philadelphia Researchers Show How Patellar Instability Affects Knee Development in Growing Youth
Researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated how trochlear anatomy evolves during pediatric development with and without recurrent patellofemoral instability (PFI), a condition where the kneecap repeatedly slips out of its groove, causing pain, swelling and difficulty with physical activity. The findings, recently reported in a landmark study in the American Journal of Sports Medicine, highlighted that PFI in growing children worsens trochlear dysplasia, which can result in further kneecap instability, while those without PFI experienced natural improvements.

Researchers at Children’s Hospital of Philadelphia Find Heart Transplant Waitlist Allocation Changes and Advancements in Clinical Care Improved Patient Outcomes for Some but Not All
Heart transplant policies underwent significant changes in 2016 and 2018, altering how hearts are allocated to patients waiting for a transplant. These changes aimed to improve survival rates and outcomes for patients on the transplant waitlist by prioritizing those with the most pressing needs. In a recent study in Circulation, researchers at Children’s Hospital of Philadelphia (CHOP) reviewed the outcomes and found some patient groups benefitted from the allocation updates, while others did not experience the same level of improvement.
Children’s Hospital of Philadelphia Preclinical Study Unveils Promising New Treatment Approach for Fanconi Anemia Patients
In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel treatment strategy for patients with Fanconi anemia (FA), a rare genetic disease resulting in loss of blood-forming stem cells in the bone marrow. The researchers successfully used in situ mRNA delivered directly into the body by lipid nanoparticles (LNPs) to target bone marrow cells and transiently restore stem cell function in patients with FA.