The thalassemias are an inherited group of heterogeneous anemias in which 1 or more of the globin chains in the hemoglobin tetramer are absent. Among the 4 classic alpha thalassemia syndromes, there ...

"This work highlights the importance of further studies to know better the consequences of genetic variability on the long-range regulation of α-globin genes in humans." - Pena et al., Molecular ...

CONVINCING evidence has been presented that the term thalassemia does not describe a specific or homogeneous entity. Bather, it refers to a group of heritable defects of hemoglobin synthesis ...

Agios Pharmaceuticals AGIO announced that its lead drug, Pyrukynd (mitapivat), an oral pyruvate kinase (PK) activator, has ...

PYRUKYND is the only medicine approved in the EU for adults with transfusion-dependent and non-transfusion-dependent alpha- or ...

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Aqvesme is expected to be available in late January 2026. The Food and Drug Administration (FDA) has approved Aqvesme™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia ...

Good day, and welcome to Agios Pharmaceuticals' Investor Conference Call and Webcast. [Operator Instructions] Please be advised that this call is being recorded at Agios' request. I would now like to ...

CHULA VISTA, Calif. — Nothing can prepare you for parenthood, and a local family was not ready to hear their unborn child may die or have life long complications. For Annemarie and Stephen, they were ...

A family of Albanian descent had an unusual form of beta thalassemia. The father of two children with mild thalassemia major had normal red-cell morphology and normal levels of hemoglobins A 2 and F, ...

CAMBRIDGE, Mass., Dec. 23, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients ...