WISKOTT-ALDRICH SYNDROME
Clinical trials for WISKOTT-ALDRICH SYNDROME explained in plain language.
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Groundbreaking Two-Transplant strategy aims to cure devastating immune diseases
⭐️ CURE ⭐️ ENROLLING_BY_INVITATIONThis study is testing a new two-step procedure for people with severe inherited immune system diseases who also have life-threatening lung damage. First, patients receive a double-lung transplant to fix their lungs. Then, they receive a bone marrow transplant from the same donor,…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:30 UTC
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Gene therapy offers hope for rare immune disorder cure
⭐️ CURE ⭐️ OngoingThis trial tests a gene therapy that aims to cure Wiskott-Aldrich Syndrome, a rare genetic disorder that weakens the immune system. Doctors collect patients' own blood stem cells, add a corrected gene in the lab, then return them to the patient. The study measures whether this tr…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE3 • Sponsor: Fondazione Telethon • Aim: ⭐️ CURE ⭐️
Last updated Mar 23, 2026 15:18 UTC
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Gentler transplant offers hope for kids with 'Bubble Boy' and other immune diseases
Disease control OngoingThis study is testing a less intense stem cell transplant procedure for children and young adults with rare immune system disorders. The goal is to see if using lower doses of chemotherapy and radiation before the transplant can help the donor cells take hold while reducing sever…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Apr 02, 2026 14:57 UTC
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New hope: rebuilding broken immune systems with donor cells
Disease control OngoingThis study is testing a treatment to rebuild the immune system in people with severe inherited immune deficiencies. Patients receive stem cells from a donor, which are meant to grow into healthy new immune cells. The goal is to control the disease and reduce life-threatening infe…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: NA • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC