T-B+ severe combined immunodeficiency due to gamma chain deficiency
MONDO:0010315Severe combined immunodeficiency (SCID) due to gamma chain deficiency, also called SCID-X1, is a form of SCID characterized by severe and recurrent infections, associated with diarrhea and failure to thrive.
Also known as: SCIDX1, T-B+ SCID due to gamma chain deficiency, T-B+ severe combined immunodeficiency due to gamma chain deficiency, T-B+ severe combined immunodeficiency, X-linked, X-linked severe combined immunodeficiency, XSCID, severe combined immunodeficiency, X-linked, X-linked recessive, SCID, X-linked
12 clinical trials for this condition and its sub-types, 10 tagged with T-B+ severe combined immunodeficiency due to gamma chain deficiency itself.
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Bubble baby breakthrough? experimental cell shot aims to reboot immune system faster
Disease control CompletedThis early-phase trial tested whether injecting special immune-building cells (HTLPs) could help children with severe combined immunodeficiency (SCID) rebuild their immune system faster after a partially matched stem cell transplant. Only 4 children took part. The goal was to see…
Phase 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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Gene therapy offers hope for 'Bubble Boy' disease, but leukemia risk remains
Disease control CompletedThis trial tested a gene therapy for children with SCID-X1, a severe immune disorder, who do not have a matched bone marrow donor. The treatment involves harvesting the child's own bone marrow cells, fixing the broken gene in the lab, and infusing the corrected cells back after m…
Phase 1/2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Gene therapy offers hope for boys with severe immune disorder
Disease control CompletedThis trial tested a gene therapy for X-linked severe combined immunodeficiency (SCID-X1), a life-threatening condition where boys are born without a working immune system. Five boys who had no matching bone marrow donor received a single infusion of their own blood stem cells tha…
Phase 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC