CHOP Hematologist Dr. Lindsey George Receives 2019 Clinical Achievement Research Award Published on Mar 06, 2019 in CHOP News Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.
Sideroblastic Anemia Is a Red-flag Sign of Single Large-scale Mitochondrial DNA Deletion Syndrome Published on Feb 21, 2019 in Children's Doctor Using the case of a 24-month-old African-American boy, Drs. Ganetzky and Goldstein discuss sideroblastic anemia and how it might be an indicator for multisystem mitochondrial disease.
Peter Kurre, MD, Appointed Director of the Comprehensive Bone Marrow Failure Center Published on Jan 18, 2019 in CHOP News Dr. Kurre will lead a world-renowned, multidisciplinary team in developing new therapies for BMF syndromes and in basic research.
CHOP Opens Manufacturing Facility, Making Tools for Disease Cures Published on Nov 01, 2018 in CHOP News 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.
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.