Precision Medicine in Rare Hematological Diseases: Pyruvate Kinase Activators

Join us on this podcast episode featuring Prof Lucia de Franceschi (Department of Medicine, University of Verona, Italy) as she discusses the latest advances on the use of Pyruvate kinase activators in the treatment of rare hematological diseases. 

Learn about the crucial role of Pyruvate kinase (PK) in erythrocyte metabolism and how pyruvate kinase activators are emerging as a new therapeutic option for chronic hemolytic anemias  that had previously no other treatment than transfusion or iron chelation. 

Discover how two oral pyruvate kinase activators, Mitapivat and Etavopivat, can help improve ineffective erythropoiesis and survival of  red cells and therefore are being tested as a new therapeutic scenario for non-transfusion dependent thalassemia patients and sickle cell disease patients. 

Discover how PK activators are able to reprogram red cells to a more normal metabolite context, which can have a beneficial effect in a wide range of rare diseases all characterized by an abnormal metabolite profile, which represents the ideal breakthrough in the management of rare diseases, as one drug has the ability to treat multiple disorders.

This is a series of podcasts covering precision medicine across several disorders, more podcasts will be available in the future. Stay tuned! 

Host: Isabel Olivera-Martinez, PhD

Guest: Prof Lucia de Franceschi

This is the seventh episode in a series of podcasts covering precision medicine across various disorders; more podcasts will be available in the future. Stay tuned!

Learn More

Matte, Alessandro, Enrica Federti, and Lucia De Franceschi. 2023. ‘Erythrocyte Pyruvate Kinase Activation in Red Cell Disorders’. Current Opinion in Hematology 30 (3): 93. https://doi.org/10.1097/MOH.0000000000000758.

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