Pyruvate Kinase Deficiency Impact Assessment (PKDIA), a weekly measure of disease impacts, measures health-related quality of life, (e.g., daily activities, concentration, physical activity and the ...

CAMBRIDGE, Mass., June 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, ...

– Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the ...

CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today ...

Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...

Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell ...

CAMBRIDGE, Mass., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined ...

CAMBRIDGE, Mass., June 28, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for ...

Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat. Topline data from ACTIVATE-Kids study of mitapivat in children who are not ...

– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for ...