Patients and families are at the heart of every rare disease journey. Guided by their experiences, we are committed to transforming care in rare disease and advancing meaningful innovation where unmet needs remain.
Each May, Brain Cancer Awareness Month highlights brain and other central nervous system cancers—some of the most complex and least understood. Despite recent advances, people with rare brain tumors still face delays in diagnosis, limited access to specialized care, and fewer treatment options when time matters most. People diagnosed with locally advanced/metastatic HER2-positive gastroesophageal adenocarcinoma continue to face limited treatment options with the need to improve outcomes. Discover why innovation in the first-line setting remains essential to improving care. For families facing diffuse midline glioma, rapid progression and scarce options have long defined the journey. Hear how following the biology, learning from outliers, and uniting researchers, clinicians, advocates, and families led to meaningful progress and a new therapeutic option for patients with H3 K27M‑mutant disease.