Most pharmaceutical and life science companies focus on genetic disorders with greater commercial potential thus, ultra-orphan diseases are often overlooked because the economics don’t add up. Our aim is to bring funding and scientific resources to the forefront so ultra-rare genetic disease research and development progresses at the same pace as other larger indications. Children with these diseases and their families urgently need access to transformative genetic medicine. Most are suffering a severely altered way of life and, in many cases, life expectancy is shorter than ten years.