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Wiskott-Aldrich Syndrome
A rare, X-linked immunodeficiency syndrome characterized by ECZEMA; LYMPHOPENIA; and, recurrent pyogenic infection. It is seen exclusively in young boys. Typically, IMMUNOGLOBULIN M levels are low and IMMUNOGLOBULIN A and IMMUNOGLOBULIN E levels are elevated. Lymphoreticular malignancies are common.
Drugs by status
Approved for this indication (1)
Phase 4 trials (1)
Phase 3 trials (1)
Phase 2 trials (22)
- plerixafor
- anti cd45 monoclonal antibody
- busulfan
- cd3,cd19 depleted hematopoietic stem cells
- eltrombopag
- tacrolimus
- naltrexone
- allogeneic hematopoietic stem cells
- romiplostim
- mycophenolate
- cyclophosphamide
- stem cells
- alemtuzumab
- Anti-thymocyte Globulin (Rabbit)
- autologous cd34 positive cells transduced with a lentiviral vector containing human was gene
- autologous cd34+ cells transduced with wasp lentiviral vector
- hematopoietic stem cell gene transfer for the wiskott-aldrich syndrome
- fludarabine
- thiotepa
- sargramostim
- melphalan
- autologous hematopoietic stem cells
Phase 1 trials (3)
Other trials (6)
Organizations
Organization Involved with Phase 4 Indications (1)
Organization Involved with Phase 3 Indications (3)
Organization Involved with Phase 2 Indications (20)
- Novartis
- GlaxoSmithKline
- Genethon
- The Foundation Institute San Raffaele G. Giglio of Cefalù
- National Institutes of Health (NIH)
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
- National Cancer Institute (NCI)
- University of Pittsburgh
- University of London
- Cornell University
- Harvard University
- Stanford University
- The Korean Society of Pediatric Hematology Oncology
- University of Florida
- University of Southern California
- Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
- State University of New York, Buffalo
- University of Paris
- Washington University in St. Louis
- Orchard Therapeutics Limited
Organization Involved with Phase 1 Indications (4)
Organization Involved with Other Experimental Indications (1)