Leukemia Sickle Cell Disease HIV Thalassemia Sickle Cell Anemia HIV and Hepatitis C Myelofibrosis Myelodysplastic Syndrome Blood Clotting Polycythemia Vera Rare Diseases Plasma Cell Disorders Sickle-Cell Disease Aplastic Anaemia Diamond Blackfan Anaemia Chronic Lymphocytic Leukemia Myelodysplastic Syndromes Cancers Chronic Myeloid Leukemia Thrombotic Thrombocytopenic Purpura Leukaemia Myelodysplastic Syndromes (MDS) Hematopoietic Disorders Aplastic Anemia Anaemia Myelodysplasia Sickle-cell Disease Beta Thalassemia Hepatitis
The mixed efficacy leaves mitapivat’s expansion prospects uncertain during an FDA review in beta thalassemia that targets a Dec. 7 decision.