A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta ...

Bone marrow transplants are increasingly being used to treat non-cancer conditions like thalassemia and sickle cell disease, ...

An essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires life-long blood transfusions for survival, non-transfusion-dependent ...

Please provide your email address to receive an email when new articles are posted on . Exagamglogene autotemcel helped patients with beta-thalassemia achieve transfusion independence. The drug also ...

Agios Pharmaceuticals has filed for regulatory approval of its drug Mitapivat (PYRUKYND) to treat adult patients with both non-transfusion-dependent and transfusion-dependent forms of alpha- or ...

Inherited blood disorders remain one of the most persistent yet under-recognised public health challenges in India. Among them, sickle cell disease and thalassemia together account for a substantial ...

I am deeply humbled by the decades of support from people around the world. It is an honor to share this painful yet ...

Beta thalassemia minor is a blood disorder that can cause mild anemia, but it often doesn't show symptoms. If both parents carry the trait for beta thalassemia minor, there's a higher risk of having a ...

Please provide your email address to receive an email when new articles are posted on . Patients with sickle cell disease and beta-thalassemia reported substantial improvements in quality of life ...