Running Toward Rare

When doctors diagnosed my 7-year-old sister with a fatal rare disease, I helped kickstart a major effort to find a cure. The charity I co-founded achieved amazing things, but my sister kept getting sicker, and somewhere along the way, I lost my will to fight. This is the story of how I found the path forward and realized we can fix not one disease, but many diseases. 1) One in 10 Americans, and 350 million people worldwide, suffer from one of 7,000 rare diseases. In many cases, these diseases have devastating consequences and zero approved treatments. They are an incredible burden on families, communities and our healthcare system. 2) Fixing all rare diseases might seem like an impossible challenge – a challenge I’ve faced for more than a decade as an advocate, non-profit leader and big sister. But if we’re brave enough to approach rare disease in a novel way, we can improve lives. Even save lives. Rare disease is everywhere. It affects us all. I hope my talk educates people on the far-reaching impact of rare disease and strategies to fix it. Most importantly, I hope it moves them to become part of the solution by supporting science and innovation or patients in their own community, or by simply having the courage to ask, What if? when faced with the world’s biggest problems.