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Leniolisib is a selective, oral small molecule inhibitor of phosphoinositide 3-kinase delta (PI3Kδ). It is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency caused by genetic variants leading to hyperactivity of PI3Kδ. Leniolisib works by blocking aberrant PI3Kδ-dependent signaling pathways in immune cells, including B and T lymphocytes, thereby reducing immune dysregulation and improving immune function. The drug has demonstrated efficacy in reducing lymphoproliferation and correcting abnormal immunophenotypes in both adult and pediatric patients with APDS[1][2][3][10].
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