Severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency
MONDO:0007064A form of SCID characterized by profound lymphopenia and very low immunoglobulin levels of all isotypes resulting in severe and recurrent opportunistic infections.
Also known as: ADA deficiency, ADA-SCID, SCID due to ADA deficiency, SCID due to ADA deficiency, early-onset, SCID due to adenosine deaminase deficiency, adenosine deaminase deficiency, adenosine deaminase deficiency, partial, Autosomal recessive, Somatic mosaicism, adenosine deaminase deficient severe combined immunodeficiency
9 clinical trials for this condition and its sub-types.
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Gentler transplant shows promise for kids with blood diseases
Disease control CompletedThis study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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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: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC