Patients with sickle cell disease and beta-thalassemia treated with exagamglogene autotemcel reported substantial and ...

Orphan drug designation incentives have helped boost enthusiasm for researching and developing drugs for beta thalassemia, a new review concludes. The review encompassed all beta-thalassemia drugs ...

Our blood is a window to our health. Doctors examine biomarkers in our blood to determine our health status. These molecules can indicate if any abnormal processes are taking place, which could point ...

Credit: Getty Images. 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 ...

A one-time CRISPR-based gene therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics now has an additional FDA approval as a treatment for the rare blood disease beta thalassemia. The ...

Agios Pharmaceuticals said a key European regulatory committee has recommended expanded approval of its Pyrukynd anemia drug for certain people with the inherited blood disorder thalassemia. Agios on ...

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Back to Healio The FDA granted priority review to ...

Thalassemia is a common genetic condition affecting red blood cells. Children with severe forms of thalassemia often develop symptoms early in life, but effective treatment can manage the condition.

- CASGEVY is the first, and only, gene editing therapy approved for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD) in Europe - - Italy has the largest ...

DEAR DR. ROACH: I am Greek and have thalassemia minor. In general, my red blood cells are much smaller than typical ones, and my iron content is significantly low. Every time I see a different doctor ...