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RUNX1 Familial Platelet Disorder
Clinical trial pipeline · Data from ClinicalTrials.gov
See which RUNX1 Familial Platelet Disorder trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which RUNX1 Familial Platelet Disorder trials you may qualify forBackground: Genes tell the body and its cells how to work. Familial platelet disease (FPD) or FPD with associated malignancies (FPDMM) is caused by a variant i…
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise eas…
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.