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John Simpkins Named Recipient of the Alex’s Lemonade Stand 2025 Pitcher of Hope Award
John Simpkins, Chief Administrative Officer of the Cancer Center at Children’s Hospital of Philadelphia, is this year’s recipient of the Alex’s Lemonade Stand Pitcher of Hope Award.
Children’s Hospital of Philadelphia Researchers Recommend New Standard of Care for Families with Hereditary Neuroblastoma Linked to ALK Mutation
Researchers at Children’s Hospital of Philadelphia (CHOP) highlighted the success of anaplastic lymphoma kinase (ALK) inhibition therapy in treating hereditary neuroblastoma, a rare subset of a common childhood cancer. Researchers suggest that the findings, published recently in JCO Precision Oncology, could help establish a new standard of care.

Children’s Hospital of Philadelphia’s First FDA Approved Gene Therapy Patient for Severe Beta Thalassemia Celebrates Two Year Treatment Anniversary
Patient no longer needs regular blood transfusions for her inherited blood disorder, and her future has been transformed.

Children’s Hospital of Philadelphia Researchers Develop Cell Atlas to Uncover Significant Shifts in the Neuroblastoma Tumor Microenvironment
Researchers at Children’s Hospital of Philadelphia (CHOP) developed a longitudinal atlas of neuroblastoma, a common and potentially deadly childhood cancer, to gain deeper understanding into precise molecular mechanisms underlying why and how certain treatments eventually become ineffective.
Children’s Hospital of Philadelphia Researchers Shed Light on How Rare Pediatric Overgrowth Syndrome May Raise Cancer Risk
New research from Children’s Hospital of Philadelphia (CHOP) offers insight into how a rare genetic condition called Beckwith–Wiedemann syndrome (BWS) affects the liver and may raise cancer risk.

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.
New Research from Children’s Hospital of Philadelphia to Transform Standard of Care Across the Globe for Newly Diagnosed Children with B-cell Acute Lymphoblastic Leukemia
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.
CHOP Named Qualified Treatment Center for Lenmeldy, the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy
CHOP’s Leukodystrophy Center, one of the leading centers of its kind in the nation, now offers Lenmeldy to treat children with metachromatic leukodystrophy (MLD).
Children’s Hospital of Philadelphia Researchers Find Pediatric Brain Tumor Survivors Are At-Risk for Social Deficits, Fewer Friendships, and Poor Peer Relationships
In a recent study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated that children treated for brain tumors face persistent social challenges, including limited friendships and reduced peer acceptance, extending into adulthood. The study is the first to link disruptions in white matter connectivity to impaired social functioning in survivors of pediatric brain tumors.
Children’s Hospital of Philadelphia Researchers Identify Gene Signature for High-Risk Form of T-Cell Acute Lymphoblastic Leukemia
Study identified a drug for potential use in future precision medicine clinical trials