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PTEN harmartoma tumor syndrome with immune disorder

MONDO:0700330

Also known as: PTEN Deficiency (LOF), PTEN harmartoma tumor syndrome with immune disorder

1 clinical trial for this condition and its sub-types.

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Broader categories

Disease (680) Hereditary disease (176) Immune system disorder (148) Immunodeficiency disease (54) Human disease (14) Disease of genetic or genomic mechanism (2) Disease by body system or component (0) Disease by etiologic mechanism (0)

Not yet finished but already full! 1

New drug aims to calm overactive immune system in rare disorders

Disease control Ongoing

This study tests a drug called leniolisib in 12 people aged 12 to 75 with certain primary immunodeficiency disorders. The goal is to see if the drug is safe and can help control immune system problems. Participants will receive three different doses of the drug, and researchers w…

Phase: PHASE2 • Sponsor: Pharming Technologies B.V. • Aim: Disease control

Last updated Jun 27, 2026 09:05 UTC