pyruvate kinase deficiency

Pyruvate kinase 是一种糖酵解酶，催化磷酸烯醇式丙酮酸和 ADP 转化为丙酮酸和 ATP。

PKR-IN-2 是丙酮酸激酶PKR 亚型的激活剂，可用于 PKR 功能相关疾病的研究，包括癌症、糖尿病、肥胖、自身免疫性疾病、良性前列腺增生等。

Mitapivat (PKR-IN-1) 是口服具有活性的变构丙酮酸激酶 (pyruvate kinase) 激活剂，其AC50=20 nM。它在广泛的 PKLR 基因型中增加酶活性、蛋白质稳定性和 ATP 水平。它有潜在的恢复 PK 缺乏的糖酵解途径活性的作用。

Mitapivat sulfate, also known as AG-348 sulfate hydrate, is an allosteric activator of Pyruvate Kinase (PK) that may be useful in the treatment of Pyruvate Kinase Deficiency.

Mitapivat sulfate anhydrous is an allosteric activator of Pyruvate Kinase (PK) that may be useful in the treatment of Pyruvate Kinase Deficiency.