MedPage Today8d
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 ...
St. Louis Post-Dispatch1mon
Dr. Roach: Low iron stores due to thalassemia call for an evaluation
You probably had a hemoglobin electrophoresis test done at some point to make the diagnosis of beta thalassemia minor, so this would be useful to present to new doctors. Making a definitive ...
Nature2y
Thalassemia: the long road from the bedside through the laboratory to the community
I owe my long interest in inherited blood diseases and tropical medicine to a series of characteristically bizarre decisions by the British Army. In 1958, 2 years after qualifying in medicine from ...
Hosted on MSN25d
Managing Non-Transfusion-Dependent Thalassemia Presents Challenges
An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival, non-transfusion-dependent ...
pharmaphorum2y
Commercialization of novel gene-editing technology in beta-thalassemia
Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Several manufacturers are developing novel treatments for the disease, including Vertex, which has partnered ...
2minutemedicine.com10d
Beti-cel gene therapy achieves high rates of transfusion independence in severe β-thalassemia
There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: ...