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Pediatric Patients Aged 1 to 6 Years With APDS

Pharming Technologies B.V. · 2023
ClinicalTrials.gov · Datasets · License: Public Domain · 2023
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This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
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