Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window In a small study ...

Iron deficiency anemia (IDA) and thalassemia represent two of the most prevalent causes of microcytic anemia worldwide. Effective screening is critical not only to guide appropriate treatment — as IDA ...

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 ...

Investigators used MRI to evaluate brain perfusion in patients with transfusion-dependent (n=54) and non–transfusion-dependent (n=23) β-thalassemia versus 56 controls. They found that relative ...

UCSF Benioff Children's Hospital Oakland is the first hospital in the West to administer a newly approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe ...

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

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 ...

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 ...

LONDON — 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 ...

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) shares climbed 19.97%, closing at $29.50, up $4.91, after the U.S. Food and Drug Administration approved AQVESME (mitapivat) as an oral treatment for ...