Pediatric Pyruvate Kinase Deficiency research hub

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

This study is looking at a new medicine called Mitapivat for children with a rare blood disorder called Pyruvate Kinase Deficiency (PKD). It aims to see if Mitapivat can help children who need regular blood transfusions, compared to a dummy medicine.