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First of Its Kind Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment
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. Handy’s Corner: Why Do We See Measles Outbreaks?
Dr. Lori Handy discusses why measles outbreaks occur and what can be done to prevent them.
Feature Article — Parents PACK 2024: The Year in Review
See how well you do answering questions about information shared in 2024 Parents PACK newsletter articles.
Vaccine News & Notes — December 2024
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.
Children’s Hospital of Philadelphia Researchers Reveal Important Themes to Improve Interactions Between Police and Black Autistic Youth
Novel study emphasizes the need for improved training to recognize key behavior differences in this patient population
Children’s Hospital of Philadelphia, Stanford Researchers Reveal and Refine New Immunotherapy Platform with Increased Potential to Target Cancer Cells
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
Children’s Hospital of Philadelphia Researchers Develop Automated Tool for Integration and Analysis of Electronic Medical Record Data for Complex Rare Disease Patients
MMFP-Tableau offers a readily generalizable solution to make research and clinical electronic health system data more accessible and impactful.

A Heartfelt Haven: The 3 South Playroom Refresh
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.

GAME CHANGER: How CHOP Has Elevated Pediatric Sports Medicine Care
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.
Children’s Hospital of Philadelphia Researchers Find Precision Medicine Treatment Delivers Strong Results for Infantile Fibrosarcoma and Other Solid Tumors
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.