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Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania revealed a powerful new bone marrow atlas that will offer the public a first-of-its-kind visual passport into the spectrum of healthy and diseased blood production.
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A multi-institutional clinical trial involving researchers from Children’s Hospital of Philadelphia (CHOP) and the Scheie Eye Institute at the University of Pennsylvania found that CRISPR-Cas9 gene editing delivered to fourteen individuals with a form of inherited blindness was safe and led to measurable improvements in vision in nearly half of the of the participants treated, including the two children treated at CHOP. The findings were published today in the New England Journal of Medicine.
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Target gene could serve as the basis of a future therapeutic intervention.
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The study identified obstacles like limited time during clinical visits, a lack of comprehensive resources, and insufficient notice from caregivers to prepare for discussions.
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Findings provide some of the first evidence of potential therapeutic targets for condition affecting thousands of patients with single ventricle congenital heart disease.
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In a landmark study, an international consortium led by researchers at CHOP published the final results of a key clinical trial of the gene therapy CASGEVY for the treatment of sickle cell disease.
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To celebrate April as National Volunteer Month, Children’s Hospital of Philadelphia and The Wawa Foundation, commemorating its 10th anniversary this year, have come together to announce the inaugural Helping Hands Community Care Challenge.
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Researchers revealed new insights into the long-term effects of bronchopulmonary dysplasia (BPD), a chronic lung disease that primarily affects premature infants, on patient health.
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The methodology developed in this study could help guide clinical trial design for many rare diseases
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The award, presented by The Chamber of Commerce for Greater Philadelphia, is the highest honor bestowed upon a business executive in Greater Philadelphia.