
Breakthrough for Young Patients: Agios Pharmaceuticals Paves New Path in Rare Blood Disorder Treatment
Agios Pharmaceuticals reports positive Phase 3 study results for mitapivat in treating pediatric pyruvate kinase (PK) deficiency. The ACTIVATE-Kids study showed a significant hemoglobin response in 31.6% of young participants treated with mitapivat, compared to 0% in the placebo group. Mitapivat improves