Existing drug shows promise as treatment for rare genetic disorder NIH researchers find new pathways towards treatment for autoimmune polyendocrine syndrome type 1.

https://www.nih.gov/news-events/news-releases/existing-drug-shows-promise-treatment-rare-genetic-disorder 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. https://www.niaid.nih.gov/news-events/existing-drug-shows-promise-treatment-rare-genetic-disorder?utm_campaign=+60793797&utm_content=&utm_medium=email&utm_source=govdelivery&utm_term=