AMED syndrome, digenic
MONDO:0030894Also known as: AMED syndrome, digenic, AMEDS, AMeD syndrome, aplastic anemia-intellectual disability-dwarfism syndrome, bone marrow failure syndrome 7, digenic
20 clinical trials for this condition and its sub-types.
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New transplant cocktail aims to cut complications in bone marrow failure
Disease control CompletedThis phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…
Phase: PHASE2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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New chemo combo may lower death risk in child stem cell transplants
Disease control CompletedThis study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…
Phase: PHASE2 • Sponsor: medac GmbH • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Drug may help bone marrow failure patients recover faster after transplant
Disease control CompletedThis study tested a drug called eltrombopag in 118 people with bone marrow failure who had a stem cell transplant. The goal was to see if the drug could help their platelet levels recover faster after the transplant. Platelets are blood cells that help stop bleeding, and slow rec…
Phase: PHASE2, PHASE3 • Sponsor: The First Affiliated Hospital of Soochow University • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC