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Primary and Safety Outcomes of a Phase 3 Open-Label, Single-Arm, 12-Week Study of Treatment with PI3Kδ Inhibitor Leniolisib in Pediatric Patients Aged 4–11 Years with Activated PI3Kδ Syndrome (APDS)

V. Rao, Manish J. Butte, Bénédicte Neven, Michaela Semeraro, Yael Gernez, Shanmuganathan Chandrakasan, Hirokazu Kanegane, Takahiro Yasumi, Kazushi Izawa, Alanvin Orpia, Ladan Foruraghi, Lauren Pauls, Lavenda Chirombo Kluczynski, Jason Bradt, Anurag Relan, Gulbu Uzel

J Hum Immun (2025) 1 (CIS2025): CIS2025abstract.6.

https://doi.org/10.70962/CIS2025abstract.6

Published: 25 April 2025

View articletitled, Primary and Safety Outcomes of a Phase 3 Open-Label, Single-Arm, 12-Week Study of Treatment with PI3Kδ Inhibitor Leniolisib in Pediatric Patients Aged 4–11 Years with Activated PI3Kδ Syndrome (APDS)