Beti-cel is an investigational one-time gene therapy that works by adding functional copies of a modified form of the beta-globin gene into a patient’s own hematopoietic stem cells.
The application included data from the phase 2 BEYOND study.
Researchers sought to describe 3 cases involving young high school athletes with sickle cell trait who died from exertional collapse.
Injectafer is an iron replacement product.
The application is supported by data from the phase 2 BEYOND study.
Researchers sought to determine whether red blood cell exchange transfusion may be beneficial for patients with sickle cell disease and COVID-19.
PillCam SB3 @HOME offers a telehealth option for direct visualization and monitoring of the small intestine.
Mitapivat is a first-in-class, oral, selective small molecule allosteric activator of pyruvate kinase-R enzymes.
The FDA noted that the application could not be approved in its present form and had requested an additional clinical study be conducted.
Researchers sought to better understand germline predisposition to aplastic anemia and how it contributes to proper diagnosis of the disease.
