From Patients to Pioneers

The Story Behind the Rare Disease Research Revolution In the world of rare disease research, the traditional model has long been investigator-led, with researchers and institutions setting the agenda. But what if we turned that model on its head? What if the people who live with these conditions—the patients themselves—could decide the research questions, lead […]

Introducing the Rare Disease Research Network

A Revolutionary Cross-sector Approach to Rare Disease Research In a world where more than 300 million people live with over 11,000 rare diseases, research that truly meets the needs of patients is paramount. Unfortunately, the journey to effective treatment for rare diseases has often been marred by prolonged diagnostic delays, limited treatment options, and insufficient […]