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Center for Precision Medicine for High-Risk Pediatric Cancer News

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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

Jan 31, 2025

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

Jan 28, 2025

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.

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