3 recruiting studies matching your filters
To learn about the safety and effects of low-dose sirolimus in participants with RUNX1-FPD.
Background: Runt-related transcription factor 1 (RUNX1) gene regulates the formation of blood cells. People with mutations of this gene may bleed or bruise ea…
To evaluate the safety and feasibility of collecting hematopoietic stem cells (HSC) in participants with RUNX1-FPD.