Synonyms AUTOIMMUNITY-IMMUNODEFICIENCY SYNDROME, X-LINKED, Autoimmune enteropathy type 1, Autoimmunity-Immunodeficiency Syndrome, X-Linked + [42] |
Introduction An X-linked recessive autoimmune condition caused by mutation(s) in the FOXP3 gene, encoding the forkhead box P3 transcription factor. The condition is characterized by infantile onset of severe diarrhea due to enteropathy, type 1 diabetes mellitus, and dermatitis. Associated features may include hypothyroidism, autoimmune hemolytic anemia, thrombocytopenia, lymphadenopathy, hepatitis, and nephritis. The condition is usually fatal before age 2 years if not treated with bone marrow transplantation. |
Target- |
Mechanism T lymphocyte replacements |
Active Org.- |
Originator Org. |
Inactive Indication- |
Drug Highest PhasePhase 1 |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target- |
Mechanism- |
Active Org. |
Originator Org. |
Inactive Indication- |
Drug Highest PhasePreclinical |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Target- |
Mechanism- |
Active Org. |
Originator Org. |
Inactive Indication- |
Drug Highest PhasePreclinical |
First Approval Ctry. / Loc.- |
First Approval Date20 Jan 1800 |
Start Date18 Aug 2023 |
Sponsor / Collaborator- |
Start Date20 Dec 2022 |
Sponsor / Collaborator- |
Start Date22 Mar 2022 |
Sponsor / Collaborator |