Mitapivat is the first FDA-approved oral therapy for anaemia in all forms of thalassemia Reduced transfusion dependence may improve long-term quality of life Thalassemia is an inherited blood disorder ...

Agios’ Pyrukynd is FDA-approved for pyruvate kinase deficiency but generated modest sales of $8.6 million in Q2 2024. Mitapivat shows promise in treating SCD and thalassemia, with ongoing Phase 3 ...

(CNN) — The US Food and Drug Administration has approved a second use for the first CRISPR-based medicine, Casgevy, which was approved in December to treat sickle cell disease. The groundbreaking ...

What Is Aqvesme, and Why Does It Matter? Aqvesme (mitapivat) is a new oral medicine approved by the FDA for treating anemia in adults with alpha- or beta-thalassemia, including those who need and ...

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

Currently, there are no clear guidelines for managing extramedullary hematopoiesis in transfusion-dependent beta thalassemia. BT, a condition characterized by reduced production of the beta-globin ...

Company will host investor conference call and webcast tomorrow, December 24 at 8:00 a.m. ET CAMBRIDGE, Mass., Dec. 23, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO), a commercial-stage ...

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 ...

On July 18th, a Memorandum of Understanding (MOU) was signed between BGI Genomics, Faculty Of Medicine Siriraj Hospital at Mahidol University, Bangkok Genomics Innovation Public Company (the BGI ...

When Gaurvik was just six months old, he was diagnosed with Beta Thalassemia Major, a serious inherited blood disorder. This meant his body couldn't make enough healthy red blood cells, and he would ...

"The multidisciplinary aspect of the guidelines meant that we really needed rigorous input from endocrinologists who have expertise in male and female infertility, as well as from obstetricians and ...