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Hemoglobinuria, Paroxysmal
A condition characterized by the recurrence of HEMOGLOBINURIA caused by intravascular HEMOLYSIS. In cases occurring upon cold exposure (paroxysmal cold hemoglobinuria), usually after infections, there is a circulating antibody which is also a cold hemolysin. In cases occurring during or after sleep (paroxysmal nocturnal hemoglobinuria), the clonal hematopoietic stem cells exhibit a global deficiency of cell membrane proteins.
Drugs by status
Approved for this indication (6)
Phase 4 trials (1)
Phase 3 trials (13)
Phase 2 trials (28)
- busulfan
- prednisone
- tacrolimus
- naltrexone
- Filgrastim
- levamisole
- lfg316
- allogeneic hematopoietic stem cells
- cyclosporine
- mycophenolate
- alemtuzumab
- Anti-thymocyte Globulin (Rabbit)
- methotrexate
- rituximab
- basiliximab
- haplo-identical hematopoietic stem cells
- sargramostim
- sodium chloride
- zilucoplan
- treosulfan
- autologous hematopoietic stem cells
- herv-e tcr transduced cd8+/cd34+ t cells in hlaa* 11:01 positive patients
- Vemircopan
- bcx9930
- kp104
- oms906
- hsk39297
- aln-cfb
Organizations
Organization Involved with Phase 4 Indications (1)
Organization Involved with Phase 3 Indications (16)
Organization Involved with Phase 2 Indications (15)
- Achillion Pharmaceuticals
- BioCryst
- Alnylam Pharmaceuticals
- Ra Pharmaceuticals
- National Cancer Institute (NCI)
- University of Arizona
- Cornell University
- Duke University
- University of Washington
- Indiana University
- Johns Hopkins University
- Radboud University
- University of Naples Frederico II
- State University of New York, Buffalo
- CMIC Co, Ltd. Japan
Organization Involved with Other Experimental Indications (1)