ADENOSINE DEAMINASE DEFICIENCY
Clinical trials for ADENOSINE DEAMINASE DEFICIENCY explained in plain language.
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Gentler transplant shows promise for kids with blood diseases
Disease control OngoingThis 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…
Matched conditions: ADENOSINE DEAMINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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Gene therapy survivors monitored for decades in new study
Knowledge-focused ENROLLING_BY_INVITATIONThis study follows 70 people who previously received gene therapy for a rare immune disorder called ADA-SCID. Researchers want to see how well the treatment worked over time and check for any long-term side effects. No new treatment is given—just regular health checkups.
Matched conditions: ADENOSINE DEAMINASE DEFICIENCY
Sponsor: University of California, Los Angeles • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC