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 ...

PYRUKYND is also approved for adults with PK deficiency in the U.S. and Europe, and a marketing application for PYRUKYND in thalassemia is currently under review by the European Commission. In the U.S ...

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 ...

Bluebird bio’s blood disorder gene therapy has flown the nest early in the U.S. Wednesday, the FDA approved the company’s one-time beta thalassemia treatment Zynteglo, also known as betibeglogene ...

While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell transplantation (HSCT) remains the only widely accessible curative option for this ...

FRANKFURT, Germany — Updated results released Friday highlighted the lasting benefits of a CRISPR-based medicine for blood disorders developed by Vertex Pharmaceuticals and CRISPR Therapeutics, as the ...

Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in ...

It costs around RM3mil to treat a thalassemia patient from infancy up to the age of 40, says Datuk Seri Dr Dzulkefly Ahmad.

Beta thalassemia and sickle cell are two red blood cell disorders which both come with massive health implications and shortened lifespans, but at least for UK-based patients the former may soon be ...

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

Health Minister Datuk Seri Dr Dzulkefly Ahmad said the estimated cost of treatment, for patients who depend on blood transfusions, reaches nearly RM3 million per patient, from childhood to the age of ...