DYSKERATOSIS CONGENITA
Clinical trials for DYSKERATOSIS CONGENITA explained in plain language.
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New transplant method aims to reduce complications in bone marrow failure patients
Disease control OngoingThis study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: NA • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New drug combo aims to make bone marrow transplants safer for kids
Disease control OngoingThis pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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New transplant approach aims to cure blood failure without harming lungs or liver
Disease control OngoingThis study tests a bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk…
Matched conditions: DYSKERATOSIS CONGENITA
Phase: PHASE2 • Sponsor: Boston Children's Hospital • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC