Introduction
Sickle cell disease (SCD) is an inherited disorder that can cause severe pain episodes, anemia, and cumulative damage to many organs, affecting long-term health and physical function, as well as quality of life. SCD is caused by the polymerization abnormal hemoglobin S, leading to sickling of red blood cells.
Mitapivat (AG-348) is an oral medication that increases the activity of the enzyme pyruvate kinase and has beneficial effects for red cell metabolism that may reduce sickling and improve anemia in individuals with SCD. We are very interested in the effect of long-term mitapivat treatment on quality of life in individuals with SCD. Therefore, we greatly appreciate your time and help with completing these health questionnaires.
To get started:
- Navigate through the menu to learn more about the study.
- Log in to the application and fill out questionnaires.
Please contact us if you have any questions or concerns.