CHOP Opens Manufacturing Facility, Making Tools for Disease Cures 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. Read more
Targeting Abnormal Signals May Be New Method to Treat a Childhood Blood Disease Published on Oct 25, 2018 in CHOP News Blocking disease-causing biological signals may lead to a new treatment for the life-threatening childhood blood disorder Fanconi anemia.
CHOP Investigator and Experimental One-dose Gene Therapy for Hemophilia B Featured in The New York Times Published on Aug 13, 2018 in CHOP News CHOP breakthrough helps scientists edge closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.
Enzyme Identified as Possible Novel Drug Target for Sickle Cell Disease, Thalassemia Published on Jul 19, 2018 in CHOP News 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.
One-dose Gene Therapy Produces Clotting Factor, Safely Stops Bleeding in Hemophilia B Patients Published on Dec 07, 2017 in CHOP News 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.
ITP Update: Immune Thrombocytopenia — Not Idiopathic and Not Purpuric Published on Jan 27, 2017 in Children's Doctor 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.
Neurological Symptoms in the Sickle Cell Population Published on Jan 27, 2017 in Children's Doctor Hematology fellow Jessica Foster, MD, reflects on the often difficult assessment of neurological symptoms in the sickle cell patient population.
Thrombophilia Testing in Healthy Children with Related Family History Published on Jan 27, 2017 in Children's Doctor 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.
Iron Deficiency Anemia: When Iron Pills Don’t Help Published on Jan 27, 2017 in Children's Doctor 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.
After One Dose of Gene Therapy Hemophilia B Patients Maintain Near-normal Levels of Clotting Factor Published on Dec 05, 2016 in CHOP News, International Update In a clinical trial led by a CHOP researcher, adult patients with hemophilia B were able to pursue daily activities without disabling bleeds.