Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, a key compound in hemoglobin. This study marks the first time researchers studied gene therapy to treat this disease, which the authors underscore could have an impact on a broad spectrum of diseases. The research was published today and featured on the cover of the journal Blood.

Dr. Lori Handy discusses why measles outbreaks occur and what can be done to prevent them.

MMFP-Tableau offers a readily generalizable solution to make research and clinical electronic health system data more accessible and impactful.

The 3 South Playroom in CHOP’s Main Building officially reopened after a renovation funded by generous support from Dancing While Cancering, the Maddie Kramer Foundation.

TRACeR-I more accurately recognizes a wide variety of surface proteins expressed by cancer cells that make them easier to target with the body’s own immune system

Three pediatric sports medicine leaders from CHOP share perspectives about how youth athletes have changed, how the field has evolved, and predictions for the future.

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Children’s Oncology Group (COG) found that larotrectinib, an oral drug that stops cancer-cell growth, was highly effective in pediatric patients with newly diagnosed infantile fibrosarcoma (IFS) or other solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation. The study, published today in the Journal of Clinical Oncology, is the first COG trial to test precision medicine in a front-line setting across all different types of solid tumors based on a genetic biomarker rather than histology. It is poised to redefine the approach to treatment for newly diagnosed fibrosarcoma and other solid tumors with the NTRK gene fusion, potentially reducing or eliminating the need for these patients to receive chemotherapy.

Novel study emphasizes the need for improved training to recognize key behavior differences in this patient population

Researchers at Children’s Hospital of Philadelphia (CHOP) and the Children’s Oncology Group (COG) announced the results of a Phase 3 study that demonstrated adding the bi-specific T-cell engager, blinatumomab, to chemotherapy for newly diagnosed National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL) pediatric patients significantly improves survival outcomes. The results were published today in the New England Journal of Medicine and will be presented at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition on December 8.

Find info about: VMP's essay contest for students, VEC's new YouTube channel and video series, a new critical thinking resource, and when politicization of science results in lack of access to healthcare resources.