Diseases Studied

The Rare Diseases Clinical Research Network is an NIH-funded research network of 20 active consortia or research groups working to advance treatment for diseases that are rare. Use the search tools on this page to find the diseases we currently study. You can reach out to the indicated consortia or research groups for more information on those diseases and studies underway.

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All Diseases > Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome (WAS)

Alternative Names: Eczema-Thrombocytopenia-Immunodeficiency Syndrome; Wiskott Syndrome (IMD2)

Disease Category: Primary Immune Deficiency Disorders

A disorder characterized by immunodeficiency (weakened immune system) due to abnormal or dysfunctional white blood cells. Symptoms include eczema (inflamed patches of red, irritated skin) and microthrombocytopenia (low levels and decreased size of platelets) which lowers blood clotting ability. Affected individuals have a high bleeding predisposition, higher risk of developing lymphoma (cancer of the immune system cells) and autoimmune disorders, such as rheumatoid arthritis, vasculitis (inflammation of the blood vessels), or hemolytic anemia (self-destruction of red blood cells).

Research groups studying this disease

Primary Immune Deficiency Disorders
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Primary Immune Deficiency Treatment Consortium (PIDTC)

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Improves the diagnosis, treatment, and quality of life of people affected by primary immunodeficiency.

Works to improve treatment, diagnosis, and find a cure for primary immunodeficiency through research, education, support, advocacy, awareness, and newborn screening.

Funds research to find improved cures for Wiskott-Aldrich syndrome and supports families living with the disease around the world.