Thursday, May 30, 2024 Existing Drug Shows Promise as Treatment for Rare Genetic Disorder Cross-sections of lungs from mice with the gene deficiency that causes APS-1, showing damaged tissue in mice not administered ruxolitinib (left) and healthy tissue in mice administered ruxolitinib (right). Black bars represent 1 mm. Credit: NIAID 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. The study, led by researchers at NIAID, was published today in the New England Journal of Medicine. Learn More National Institute of Allergy and Infectious Diseases  |  National Institutes of Health

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