WISKOTT-ALDRICH SYNDROME
Clinical trials for WISKOTT-ALDRICH SYNDROME explained in plain language.
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Gene therapy breakthrough offers hope for rare immune disorder
Disease control OngoingThis study tests a gene therapy for people with Wiskott-Aldrich syndrome, a rare genetic disorder that weakens the immune system and causes bleeding. The treatment uses the patient's own blood stem cells, which are modified in a lab to fix the faulty gene, then returned to the bo…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE3 • Sponsor: Fondazione Telethon • Aim: Disease control
Last updated May 17, 2026 03:00 UTC
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New hope for kids with 'Bubble Boy' disease: stem cell transplant study
Disease control OngoingThis study tests a stem cell transplant (bone marrow transplant) for people with severe immune system problems, like SCID (bubble boy disease) and Wiskott-Aldrich syndrome. The goal is to replace the faulty immune system with healthy donor cells. 57 participants will be followed …
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: NA • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated May 17, 2026 02:58 UTC
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Milder stem cell transplant offers hope for kids with rare immune diseases
Disease control OngoingThis study tests a stem cell transplant for children and young adults up to age 28 with serious immune system disorders. The goal is to replace their faulty immune cells with healthy donor cells using a milder chemotherapy-like preparation to reduce side effects. The study tracks…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated May 14, 2026 12:02 UTC
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Double transplant offers hope for kids with rare immune diseases
Disease control ENROLLING_BY_INVITATIONThis study tests a new approach for people aged 5 to 45 with primary immune disorders and severe lung damage. Participants receive a double lung transplant followed by a stem cell transplant from the same donor. The goal is to see if this combination is safe and can help the body…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated May 07, 2026 18:41 UTC