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.

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

A mother has told of her heartbreak at losing both her sons to mitochondrial disease, and her attempts to raise awareness of ...

Genes involved in export of lipooligosaccharides, heme (used in membrane-bound succinate dehydrogenase), and other components related to the cell membrane or extracellular structures were detected ...

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.