Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …

One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease: Follow-Up Results of a Phase
2, Open-Label Study Page 1 One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease …

Myrthe J. van Dijk1,2,*, Minke AE Rab2,3, Brigitte A. van Oirschot2, Jennifer Bos2, Cleo
Derichs1, Anita W. Rijneveld3, Marjo Page 1 ASH2023 One-Year Safety and Efficacy of …

Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …

Sickle cell disease (SCD) is a hereditary red blood cell (RBC) disorder that affects millions of
people worldwide. 1, 2 SCD is caused by a single nucleotide mutation in the HBB gene (c …

Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …

Targeting the primary pathogenic event of sickle cell disease (SCD), polymerization of sickle
hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral pyruvate …

Sickle cell disease (SCD) is a hereditary red blood cell (RBC) disorder that affects millions of
people worldwide. 1, 2 SCD is caused by a single nucleotide mutation in the HBB gene (c …

Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …

Targeting the primary pathogenic event of sickle cell disease (SCD), the polymerization of
sickle hemoglobin (HbS), may prevent downstream clinical events. Mitapivat, an oral …