Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these diso…

Diamond Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+2 more

~Estimated

~$10 – $675

National Cancer Institute (NCI)NCT00027274

Phase 2

Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588

Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the s…

Acute LeukemiaSevere Aplastic AnemiaNon-hodgkin Lymphoma+6 more

~Estimated

~$10 – $7,500

Mitchell CairoNCT04099966

Mobilization of CD34+ Peripheral Blood Stem Cells With Filgrastim (Granulocyte-colony Stimulating Factor) and Plerixafor From Patients With Diamond Blackfan Anemia Syndrome

Gene therapy is a new possible treatment for the anemia of DBAS. Gene therapy will soon be available for patients with RPS19-mutated DBAS. This involves insert…

Diamond Blackfan Anemia

~Estimated

~$10 – $1,125

Northwell HealthNCT07186179