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Phase 3 trial of leniolisib in activated PI3Kδ syndrome, dabrafenib plus trametinib in relapsed/refractory hairy cell leukemia, and TγδLGL leukemia as a more symptomatic subset of leukemia

Blood Podcast

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APDS - A Phase 3 Study in a Rare Disorder

Study found that both co-primary endpoints were met. Laniolicib significantly increased the percentage of naive B cells in peripheral blood at day 85 from baseline, with a difference in the adjusted mean change of 37.3%. Five patients reported serious adverse events, but none were felt to be related to the study medication and there were no deaths.

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