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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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
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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 disorders where the immune system doesn't work properly. The goal is to use a milder treatment to help the patient's body accept the donor's healthy immune cells with fewer…
Matched conditions: WISKOTT-ALDRICH SYNDROME
Phase: PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Mar 23, 2026 15:29 UTC