Researchers sought to determine whether a novel approach to haploidentical bone marrow transplantation would be effective in patients with aplastic anemia.
Researchers sought to evaluate the efficacy and safety of mitapivat in adults with pyruvate kinase deficiency receiving regular transfusions.
Researchers sought to determine whether children with SCD who undergo HSCT would experience normalization of whole-brain hemodynamics.
Researchers sought to determine whether C3 or C5 blockade could be beneficial for preventing breakthrough hemolysis in patients with PNH.
Researchers sought to determine outcomes of transplantation in relapsed/refractory severe anemia with the use of a haploidentical donor.
Researchers sought to determine whether outcomes would improve with preoperative transfusion prior to cholecystectomy in patients with SCD.
KP104 is a bifunctional, first-in-class biologic that works by selectively inhibiting the alternative and terminal pathways in the complement system.
Researchers sought to determine whether silent cerebral infarctions were associated with cognitive performance or impairment among patients SCA.
Researchers sought to develop a protocol to detect and quantify PNH in erythrocytes and leukocytes.
Researchers sought to determine whether inhibiting the terminal complement pathway would be beneficial in treating patients with PNH.
