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.

Preclinical study suggests immunotherapies targeting mitochondria could treat certain tumors.

CHOP is recruiting research participants for a new observational study called, “DISCOVERY of Risk Factors for Type 2 Diabetes in Youth,” led by Lorraine L. Katz, MD.

Tzield, the first FDA-approved treatment to delay the onset of clinical diabetes, is now available in the Type 1 Diabetes clinic at CHOP

Learn more about how genetic testing offers the best means of distinguishing between the focal and diffuse forms of HI in children.

Aimee Ortega describes her thoughts participating in the Global Health Allies Program in the Dominican Republic.

Researchers at Children’s Hospital of Philadelphia (CHOP) recently reviewed best practices, guidelines, and care path objectives for early-onset sepsis (EOS), an invasive bacterial or fungal infection that occurs in infants within the first week of life and can be fatal. Their recommendations were recently published in Clinics in Perinatology.

Check out several resources that may be helpful for your vaccine-related efforts, including abstract submission opportunity, infodemic management courses, updated vaccine administration resources, and more.

An interprofessional team at Children’s Hospital of Philadelphia is transforming CHOP’s approach to fetal medicine through a first-of-its-kind data integration platform. The Clinical Outcomes Data Archive (CODA) platform offers clinicians real-time data to help inform earlier interventions and treatment strategies in CHOP’s Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT). A paper outlining CODA’s benefits, applications and potential broader impact was recently published in the journal Fetal Diagnosis and Therapy.

Find out about the topics discussed in a comprehensive analysis of misinformation in science published by the National Academies of Science.