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Clinical Trials for Congenital Bone Marrow Failure Syndromes

• Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
• 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
• Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT