Approved Drug Reduces Symptoms of Rare Genetic Syndrome

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 the National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID), was published May 29, 2024 in the New England Journal of Medicine. The article is titled “The Role of Interferon-γ in Autoimmune Polyendocrine Syndrome Type 1.”
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