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

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

IHMT-PI3Kδ-372

PI3Kδ inhibitors(Merck)

PI3Kδ/V-IN-01

ALPORT SYNDROME FOUNDATION

United States

RETT SYNDROME RESEARCH TRUST

United States

International Rett Syndrome Foundation

United States

Pharming Group Reports Positive Phase II Topline Data for Leniolisib in PIDs

Pharming Advances Rare Disease Pipeline with Napazimone for Mitochondrial Disease and Leniolisib Expansion

Pharming Group Initiates Phase II Trial of Leniolisib for Primary Immunodeficiencies