Model‐informed drug development of voxelotor in sickle cell disease: Exposure‐response analysis to support dosing and confirm mechanism of action

• Autores principales: Michelle L. Green (Autor), Radojka M. Savic (Autor), Margaret Tonda (Autor), Karin Jorga (Autor), Carla B. Washington (Autor)
• Formato: Libro
• Publicado: Wiley, 2022-06-01T00:00:00Z.
• Materias: article
• Acceso en línea: Connect to this object online.

Abstract Sickle cell disease (SCD) is characterized by the production of sickle hemoglobin (HbS), which when deoxygenated, polymerizes leading to red blood cell damage and hemolytic anemia, a defining feature of SCD. Voxelotor (Oxbryta) is a small molecule inhibitor of HbS polymerization that disrup...