Explore this collection of Stanford technologies that offer hope for individuals affected by rare diseases such as IPEX, Duchenne's muscular dystrophy, Friedreich's Ataxia, polycythemia, and others. This innovative portfolio encompasses cutting-edge cell & gene therapies, which harness the power of genetic engineering and cellular manipulation to address the root causes of these conditions. Additionally, the collection features advanced small molecule & antibody therapies that precisely target disease pathways, providing novel treatment options for patients in need.