SEVERE COMBINED IMMUNODEFICIENCY DUE TO RAG1 DEFICIENCY
Clinical trials for SEVERE COMBINED IMMUNODEFICIENCY DUE TO RAG1 DEFICIENCY explained in plain language.
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Gene therapy offers hope for 'Bubble Boy' disease without a donor
Disease control TerminatedThis trial tests a gene therapy for infants under 2 years old with RAG1-deficient severe combined immunodeficiency (SCID), a life-threatening condition where the immune system barely works. The therapy uses the child's own blood stem cells, modified with a corrected gene, to try …
Matched conditions: SEVERE COMBINED IMMUNODEFICIENCY DUE TO RAG1 DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Videja B.V. • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: SEVERE COMBINED IMMUNODEFICIENCY DUE TO RAG1 DEFICIENCY
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC