30
Participants
Start Date
December 1, 2017
Primary Completion Date
November 30, 2022
Study Completion Date
November 30, 2023
Rapamycin
Gain-of-function (GOF) PIK3CD mutations lead to PI3Kδ hyperactivity, with the downstream mediators Akt and mTOR hyperphosphorylated. The mammalian/mechanistic target of rapamycin inhibitor rapamycin may be effective to control the progress of this disease.
Children's Hospital of Fudan University, Shanghai
Children's Hospital of Fudan University, Shanghai
Children's Hospital of Fudan University
OTHER