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– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH – – Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks – BOSTON…

Key Points Donor-derived T cells with native specificity for multiple myeloid leukemia antigens can be expanded ex vivo. Infusion of mLSTs after HCT is well tolerated and produces antileukemia effects. Abstract Relapse after allogeneic hematopoietic stem cell transplantation (HCT…

Meningioma-1 (MN1) is a protein whose overexpression has been linked to acute myeloid leukemia (AML) and brain tumors, and 70% to 80% of AML patients with high overexpression of MN1 die within two years. Yet the mechanism by which MN1 causes these diseases has largely remained a…

The novel agentSY-1425 combined with azacitidine appears to be active in retinoic acid receptor alpha (RARA) superenhancer–positive newly diagnosed and relapsed or refractory acute myeloid leukemia (AML), according to the results of a phase II trial presented at the 2020 American…