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 ...

Findings indicate that altering glucose metabolism in T cells boosts their therapeutic potential against melanoma, paving the way for improved immunotherapies.

A Prescription Drug User Fee Act target date of May 27, 2025 has been assigned to the application. The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application ...

DCA will be used with a proprietary genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD) 1. The FDA granted Priority Review for the NDA and ...

Bioengineering and Environmental Sciences Lab, Department of Energy and Environmental Engineering, CSIR-Indian Institute of Chemical Technology (CSIR-IICT), Hyderabad 500007, India ...

Engineering Research Center of Ministry of Education on Food Synthetic Biotechnology and School of Biotechnology, Jiangnan University, 1800 Lihu Road, Wuxi, Jiangsu 214122, China Science Center for ...

The purpose of this study was to investigate whether circulating pyruvate kinase M2 (PK‐M2) levels are elevated in the peripheral blood and to assess their association with diagnosis and prognosis in ...

The intact structure of mammalian multi-enzymatic pyruvate dehydrogenase complex (PDHc) remains poorly understood. Through in-situ structural analysis, the authors unveil its intrinsic dynamics ...