"Existing Drug Offers Hope for Rare Genetic Disorder"
NIH researchers have found that ruxolitinib, a drug approved for certain autoimmune diseases and cancers, can alleviate symptoms of autoimmune polyendocrine syndrome type 1 (APS-1), a rare genetic disorder. The study, published in the New England Journal of Medicine, showed that elevated levels of interferon-gamma (IFN-gamma) are linked to APS-1, and ruxolitinib helps normalize these levels, reducing symptoms in both mice and human participants. Further research with a larger patient group is needed to confirm these findings.