New Biomarker-based Strategy to Screen and Monitor for Activated Phosphoinositide 3-kinase δ Syndrome

计划入组 14 人 · 首次发布于 2024 年 11 月 19 日

Safety, Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Repeat Doses of Inhaled Nemiralisib in Patients With APDS/PASLI

计划入组 5 人 · 首次发布于 2015 年 11 月 1 日

Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome

计划入组 30 人 · 首次发布于 2017 年 12 月 26 日

Leniolisib(Joenja)

C₂₁H₂₅F₃N₆O₂

AMP-activated protein kinase inhibitors (Generian)

Abelson tyrosine kinase inhibitors (Down syndrome/Fragile X syndrome)

ALPORT SYNDROME FOUNDATION

United States

Algemeen Ziekenhuis Delta

Belgium

International Rett Syndrome Foundation

United States

First European Patient Receives Breakthrough Treatment for Ultra-Rare APDS Immune Disorder

Pharming Group Initiates Phase II Trial of Leniolisib for Primary Immunodeficiencies

TC BioPharm to Explore Gamma Delta T-Cell Therapy TCB008 for Mpox Treatment