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Hematology/Oncology, biology, and treatment of bone marrow failure disorders, hereditary red blood cell disorders-clinical trials.
Pyruvate Kinase Deficiency Natural History Study
The purpose of this study is to describe the range and incidence of symptoms, treatments, and
complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all
ages with known PKD or with a hemolytic anemia and a family member with PKD. The study will
collect retrospective medical history, routine clinical care data, and quality of life
measures at baseline and annually for patients with PKD.
Stanford is currently not accepting patients for this trial.
For more information, please contact Heather Hilmoe, CRA, 650-725-1662.
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A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of
different dose levels of AG-348 (mitapivat) in participants with PK deficiency.