Non-transfusion-dependent thalassemia encompasses three clinically distinct forms -- beta-thalassaemia intermedia, hemoglobin ...

After a long struggle, and with the help of Frank Vella, a biochemist from Singapore University, we found that this child had thalassemia, an inherited blood disease that was thought at the time ...

Patients with beta-thalassemia major (also known as Cooley's anemia) have a reduction or absence of beta protein in their hemoglobin, resulting in severe anemia, which requires regular blood ...

The sNDA is supported by data from the phase 3 ENERGIZE and ENERGIZE-T studies, which evaluated mitapivat vs placebo in patients with NTD and TD alpha- or beta-thalassemia, respectively.

Last year, the U.K.'s National Health System (NHS) began offering stem cell transplants to adults with transfusion-dependent thalassemia for the first time. An evidence review from the NHS ...

FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and ...

If approved, AGIO's Pyrukynd will be the first oral therapy for all thalassemia subtypes. A final decision is expected by Sep. 7.