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Anemia, Diamond-Blackfan
A rare congenital hypoplastic anemia that usually presents early in infancy. The disease is characterized by a moderate to severe macrocytic anemia, occasional neutropenia or thrombocytosis, a normocellular bone marrow with erythroid hypoplasia, and an increased risk of developing leukemia. (Curr Opin Hematol 2000 Mar;7(2):85-94)
Drugs by status
Approved for this indication (1)
Phase 4 trials (1)
Phase 3 trials (7)
Phase 2 trials (25)
- methylprednisolone
- bpx-501
- cd3,cd19 depleted hematopoietic stem cells
- daclizumab
- eltrombopag
- tacrolimus
- naltrexone
- hemopoietic stem cell
- hydroxyurea
- leucine
- allogeneic hematopoietic stem cells
- cyclosporine
- mycophenolate
- sotatercept
- autologous umbilical cord blood
- cd45-depleted allogenic peripheral blood stem cell
- methotrexate
- autologous mesenchymal stem cells
- rituximab
- thiotepa
- melphalan
- trifluoperazine
- treosulfan
- autologous hematopoietic stem cells
- allogeneic transplant donor lymphocytes, depleted of tcr-αβ t cells and b cells; enriched for nk cells and tcrγδ t cells
Phase 1 trials (2)
Other trials (2)
Organizations
Organization Involved with Phase 4 Indications (1)
Organization Involved with Phase 3 Indications (2)
Organization Involved with Phase 2 Indications (18)
- Bellicum Pharmaceuticals
- Sponsor GmbH
- National Institutes of Health (NIH)
- Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache
- National Cancer Institute (NCI)
- National Heart, Lung, and Blood Institute (NHLBI)
- University of Pittsburgh
- Duke University
- New York Medical College
- Northwell Health
- Stanford University
- University of Florida
- University of Louisville
- University of Southern California
- University of California, San Francisco
- Feinstein Institute for Medical Research
- State University of New York, Buffalo
- University of Alabama, Birmingham
Organization Involved with Phase 1 Indications (1)
Organization Involved with Other Experimental Indications (2)