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Existing Drug Shows Promise as Treatment for Rare Genetic Disorder

May 30, 2024

A drug approved to treat certain autoimmune diseases and cancers successfully alleviated symptoms of a rare genetic syndrome called autoimmune polyendocrine syndrome type 1 (APS-1). Researchers identified the treatment based on their discovery that the syndrome is linked to elevated levels of interferon-gamma (IFN-gamma), a protein involved in immune system responses, providing new insights into the role of IFN-gamma in autoimmunity.

NIH Researchers Discover New Autoinflammatory Disease, Suggest Target for Potential Treatments

March 28, 2023

Scientists have identified an autoinflammatory disease caused by mutations in the LYN gene, an important regulator of immune responses in health and disease.

NIH Launches Study of Extra COVID-19 Vaccine Dose in People with Autoimmune Disease

August 27, 2021

The National Institutes of Health has begun a clinical trial to assess the antibody response to an extra dose of an authorized or approved COVID-19 vaccine in people with autoimmune disease who did not respond to an original COVID-19 vaccine regimen. The trial also will investigate whether pausing immunosuppressive therapy for autoimmune disease improves the antibody response to an extra dose of a COVID-19 vaccine in this population.

Gene Therapy Restores Immune Function in Children with Rare Immunodeficiency

May 11, 2021

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health.