The US Food and Drug Administration has approved two new breakthrough therapies for patients with sickle cell disease (SCD), and we believe both may enhance the treatment of our patients here at CHOP.
Read morePublished on in CHOP News
Stefano Rivella, PhD, an expert in creating the next-generation lentiviral vectors for the cure of hemoglobinopathies including sickle cell disease (SCD), as well as a leader in the discovery of erythroid and iron disorders including beta-thalassemia, has led three new published studies that make significant contributions to the scientific body of literature regarding red blood cells, and how manipulating the production of red cells can lead to the development of new disease-modifying therapies.
Published on in CHOP News
Dr. Oved brings deep expertise to the group, having completed scientific and clinical training that included tenures as a Fulbright Fellow with the Weizmann Institute in Israel, the NYU School of Medicine with two years as a Howard Hughes Medical Fellow, followed by a residency in Pediatrics at NYP Weill Cornell Medical College.
Published on in CHOP News
Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.
Published on 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.
Published on in CHOP News
Dr. Kurre will lead a world-renowned, multidisciplinary team in developing new therapies for BMF syndromes and in basic research.
Published on 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.
Published on in CHOP News
Blocking disease-causing biological signals may lead to a new treatment for the life-threatening childhood blood disorder Fanconi anemia.
Published on in CHOP News
CHOP breakthrough helps scientists edge closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.
Published on 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.