Inherited Bone Marrow Failure Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency [Phase 1]
This clinical trial update describes an ongoing Phase 1 study evaluating imatinib to increase RUNX1 activity in individuals with germline RU…
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies [Phase 2]
Phase 2 pilot study to assess feasibility of using decitabine with filgrastim as post-transplant prophylaxis in children and young adults wi…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Clinical trial aiming to define the natural history of squamous cell carcinoma in people with Fanconi anemia and inherited bone marrow failu…
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CLINICAL_TRIALS research · 2024-11-07Clinical Trial Update: Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study)
A multicenter clinical study at the Peter MacCallum Cancer Centre to diagnose and characterize inherited bone marrow failure syndromes and i…