Familial hemophagocytic lymphohistiocytosis 3

MONDO:0012146

Any genetic hemophagocytic lymphohistiocytosis in which the cause of the disease is a mutation in the UNC13D gene.

Also known as: FHL3, HLH3, HPLH3, UNC13D genetic hemophagocytic lymphohistiocytosis, familial hemophagocytic lymphohistiocytosis type 3, genetic hemophagocytic lymphohistiocytosis caused by mutation in UNC13D, hemophagocytic lymphohistiocytosis, familial, type 3, Hlh3

42 clinical trials for this condition and its sub-types, 1 tagged with Familial hemophagocytic lymphohistiocytosis 3 itself.

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Where it sits in the disease tree

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Part of

↑ Hereditary hemophagocytic lymphohistiocytosis (16)

Including sub-types (42) Tagged with Familial hemophagocytic lymphohistiocytosis 3 (1)

Not yet recruiting 1

Gene therapy could replace bone marrow transplants for rare immune disease

Disease control Not yet recruiting

This trial tests a gene therapy for FHL3, a rare immune disorder caused by a gene mutation. Instead of a donor stem cell transplant, patients receive their own stem cells that have been corrected with a healthy gene. The goal is to restore immune function while avoiding transplan…

Phase 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control

Last updated Jun 27, 2026 12:36 UTC

Familial hemophagocytic lymphohistiocytosis 3

Where it sits in the disease tree

Gene therapy could replace bone marrow transplants for rare immune disease