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 ...

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application for SL1009 (sodium dichloroacetate oral solution) for the treatment of pyruvate dehydrogenase complex ...

A century ago, iodine deficiency affected kids across large swaths of the country. It essentially disappeared after some food makers started adding it to table salt, bread, and some other foods ...

Pyruvate kinase (PK) deficiency is the most frequent red cell enzyme deficiency that causes hemolysis with a prevalence of 5:100,000 in most populations. 1 Association of PK deficiency with ...

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 ...

DCA will be used with a proprietary genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD ... therapy that inhibits Pyruvate Dehydrogenase ...

Vitamin deficiency due to hair loss (alopecia) may occur with deficiencies in vitamins B, D, C, and E, as well as with certain minerals like iron, selenium, and zinc. The complex roles of nutritional ...