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Pyruvate Kinase Deficiency research hub
2 indexed studies · 1 currently recruiting
- enrolling by invitationPHASE4
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
This study allows patients who are already benefiting from a drug called mitapivat in a previous trial to continue taking it. It's for those with conditions like Pyruvate Kinase Deficiency, Sickle Cell Disease, or Thalassemia, to ensure they can keep receiving the treatment if their doctor believes it's helping them.
Belgium · Brazil · Canada - active not recruiting
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is gathering information worldwide about a rare blood condition called Pyruvate Kinase Deficiency (PKD). We want to understand how it affects people over time and the treatments they receive, to help improve care in the future.
United States · Canada · Czechia