New treatment developed by CHOP researchers poised to improve sepsis outcomes.

The US Food and Drug Administration has granted accelerated approval to two novel drugs to treat sickle cell disease.

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.

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.

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.

Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.

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.

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.

Blocking disease-causing biological signals may lead to a new treatment for the life-threatening childhood blood disorder Fanconi anemia.

CHOP breakthrough helps scientists edge closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.