"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 ...

Bristol-Myers Squibb (NYSE:BMY) reported positive top-line Phase 2 results for Reblozyl in alpha-thalassemia related anemia, ...

PRINCETON, N.J., February 23, 2026--(BUSINESS WIRE)--Bristol Myers Squibb (NYSE: BMY) today announced positive top-line results from the ongoing, ex-US, Phase 2 registrational study (NCT05664737) ...

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 ...

The Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adults with non-transfusion-dependent (NTD) and ...

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 ...

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 ...

Thanks to groundbreaking in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a ...

(RTTNews) - Biopharmaceutical company Agios Pharmaceuticals, Inc. (AGIO) announced Monday that the Emirates Drug Establishment (EDE) of the United Arab Emirates (UAE) has approved PYRUKYND (mitapivat) ...

Avanzanite entered an exclusive partnership with Agios in June 2025 to manage PYRUKYND commercialization across Europe European Commission will now review the CHMP’s opinion, with the final decision ...