Advancements in Pyruvate Kinase Deficiency and Congenital Dyshaemopoietic Anemias

This chapter explores recent developments in managing pyruvate kinase deficiency, including the creation of an international patient registry that improves understanding of the disease. It also discusses emerging treatments and gene therapy trials, along with the complexities surrounding congenital dyshaemopoietic anemias and the need for better therapeutic strategies.