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HELIOS deficiency

MONDO:0800139

A non-severe combined immunodeficiency caused by a loss-of-function variation in the IKZF2 gene that is characterized by recurrent upper respiratory infections, thrush and mucosal ulcers, and chronic lymphadenopathy.

1 clinical trial for this condition and its sub-types, 0 tagged with HELIOS deficiency itself.

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Where it sits in the disease tree

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Part of

↑ Non-SCID combined immunodeficiency (1)

Including sub-types (1) Tagged with HELIOS deficiency (0)

New stem cell method aims to protect kids with rare immune disease

Disease control Stopped early

This study tested a way to improve stem cell transplants for children with combined immunodeficiency (CID), a condition where the immune system doesn't work properly. The approach removed certain naive cells from the donor stem cells to try to prevent graft-versus-host disease (a…

Phase 2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control

Last updated Jun 27, 2026 12:02 UTC