THE hemolytic anemia associated with erythrocyte pyruvate kinase deficiency is of variable severity 1–17 and probably of variable cause. In some cases severe aberrations of erythrocyte shape ...

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

Agios' mitapivat met its primary endpoint in a Phase 3 trial for PK deficiency in children. The company plans a marketing application based on the results.

Pyrukynd is currently approved by the FDA to treat adults with pyruvate kinase deficiency, a rare and debilitating blood disorder. The drug is currently the sole marketed product in the company ...

USA-based Agios Pharmaceuticals has announced that the ACTIVATE-Kids Phase III study of mitapivat in children aged 1 to <18 ...

DCA is a targeted therapy that inhibits Pyruvate Dehydrogenase Kinase (PDK) to stimulate residual Pyruvate Dehydrogenase Complex (PDC) activity and increase energy (ATP) production by mitochondria.

Presented Phase 1 clinical results on tebapivat (AG-946) in patients with sickle cell disease at ASH 2024. Pediatric Pyruvate Kinase (PK) Deficiency: Reported in a separate press release today, ...

Feb. 13, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK ... 1 to <18 years with PK deficiency who are not regularly ...