National Academies of Sciences%2c Engineering%2c and Medicine4mon
Strategic Plan and Blueprint for Addressing Thalassemia in the United States
An ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will develop a strategic plan and blueprint for addressing thalassemia in the United States. In conducting its work ...
MedPage Today7d
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent beta-thalassemia in August 2022, and exagamglogene autotemcel (exa-cel ...