Thalassemia is primarily categorized into alpha and beta variants, with beta-thalassemia commanding the largest market share due to its higher prevalence and severity. Treatment modalities include ...

Two main types of thalassemia exist, alpha and beta, depending on which part of the hemoglobin molecule is affected. Each condition further classified as mild, intermediate, or severe. Many infants ...

Presented positive results from the ENERGIZE-T Phase 3 randomized clinical trial evaluating mitapivat versus placebo in adults with transfusion-dependent alpha- or beta-thalassemia at the 66ᵗʰ ...

The sNDA is supported by data from the phase 3 ENERGIZE and ENERGIZE-T studies, which evaluated mitapivat vs placebo in patients with NTD and TD alpha- or beta-thalassemia, respectively.

CAMBRIDGE, Mass., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare ...

FDA Accepts Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia ...

Most people with thalassemia minor (alpha or beta) do not develop fatigue due to anemia, as the anemia is quite mild. If you have fatigue with exercise, I’d be concerned that you have more than ...

Most people with thalassemia minor (alpha or beta) do not develop fatigue due to anemia, as the anemia is quite mild. If you have fatigue with exercise, I’d be concerned that you have more than ...

A family of Albanian descent had an unusual form of beta thalassemia. ... De Michele D, et al: A possible case of alpha-beta thalassemia. Acta Genet (Basel) 12:202–208, 1962. PubMed.

Alpha thalassemia. Four genes are responsible for making the alpha protein. You get two from each parent. ... Beta thalassemia. Two genes are needed to make the beta protein.

Agios is hoping for a broad label in thalassemia, covering both alpha and beta subtypes regardless of transfusion dependence. They estimate that this market (18+) is 6,000 in the US.