How gene therapy for sickle cell disease changed Marie's life. www.chop.edu/centers-programs...
In a transformative moment for patients with sickle cell disease, and after rigorous clinical trials that took place at Children’s Hospital of Philadelphia (CHOP) and other sites, the Food and Drug Administration (FDA) approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent sickle cell disease pain crises. CHOP is a Qualified Treatment Center offering LYFGENIA, manufactured by bluebird bio, and also plans to offer CASGEVY, which is manufactured by Vertex Pharmaceuticals.
This video tells the story of Marie-Chantal, a participant in the clinical trials at Children's Hospital of Philadelphia.
7 дек 2023
