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The grand opening of CHOP’s new Clinical Manufacturing Facility highlights its role in manufacturing tools for 21st century precision medicine, to bring innovative treatments to patients.
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Blocking disease-causing biological signals may lead to a new treatment for the life-threatening childhood blood disorder Fanconi anemia.
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CHOP breakthrough helps scientists edge closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.
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Researchers have found a key protein that could become a target for a new treatment for sickle cell disease. In lab tests, blocking the protein reduces sickling that distorts red blood cells.
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A team of gene therapy researchers has reported on a highly active gene variant that achieved sustained factor levels in 10 adults, with no adverse effects.
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Examine the case of a 20-month-old patient diagnosed with immune thrombocytopenia after presenting with a red, pinpoint rash and a recent history of bruising.
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Hematology fellow Jessica Foster, MD, reflects on the often difficult assessment of neurological symptoms in the sickle cell patient population.
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Read about the prevalence of inherited thrombophilias (IT), potential benefits and limitations of IT testing in healthy children, and recommendations from Children Hospital's experts.
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In the pediatric population, toddlers and adolescent girls are most commonly affected by iron deficiency anemia (IDA). This case study explores when IDA should prompt investigation for malabsorption or bleeding.
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In a CHOP-led clinical trial, children with the autoimmune blood disorder ALPS had a durable, complete response, with very few side effects.