Severe Combined Immunodeficiency
Group of rare congenital disorders characterized by impairment of both humoral and cell-mediated immunity, leukopenia, and low or absent antibody levels. It is inherited as an X-linked or autosomal recessive defect. Mutations occurring in many different genes cause human Severe Combined Immunodeficiency (SCID).
Subtype terms (1)
More specific conditions that are subtypes of Severe Combined Immunodeficiency in the MeSH hierarchy.
- X-Linked Combined Immunodeficiency Diseases
23 drugs (12 approved, 11 experimental)
Drugs by status
Approved for this indication (3)
Phase 3 trials (6)
Phase 2 trials (22)
- plerixafor
- busulfan
- tacrolimus
- naltrexone
- lymphocyte immune globulin, anti-thymocyte globulin (equine)
- Filgrastim
- allogeneic hematopoietic stem cells
- sirolimus
- mycophenolate
- allogeneic lymphocytes
- alemtuzumab
- Anti-thymocyte Globulin (Rabbit)
- allogeneic cord blood
- efs-ada transduced cd34+ bone marrow cells
- allogeneic bone marrow
- unrelated pbsc with t cell depletion
- thiotepa
- sargramostim
- melphalan
- autologous hematopoietic cells transduced with a lentiviral vector with dclre1c
- autologous hematopoietic stem cells
- autologous cd34+ hematopoietic stem cells transduced ex-vivo with a gamma-globin lentiviral vector
Phase 1 trials (6)
Other trials (5)
Organizations
Organization Involved with Phase 3 Indications (6)
Organization Involved with Phase 2 Indications (24)
- Fondazione Telethon
- National Institutes of Health (NIH)
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institute of Allergy and Infectious Diseases (NIAID)
- University of Pittsburgh
- Baylor University
- University of London
- Harvard University
- Emory University
- Institut National de la Santé Et de la Recherche Médicale, France
- University of Florida
- University of Southern California
- University of California, San Francisco
- Food and Drug Administration
- Paris Descartes University
- State University of New York, Buffalo
- Vita-Salute University of Milano. Italy
- Washington University in St. Louis
- George Washington University
- California Institute for Regenerative Medicine
- Assisi Foundation
- Assistance Publique - Hôpitaux de Paris
- Children's Hospital Los Angeles
- St. Jude Children's Hospital
Organization Involved with Phase 1 Indications (5)
Organization Involved with Other Experimental Indications (2)