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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.

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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.

Patients and families cutting ribbon
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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.

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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.

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