Why We're Here

Our vision is a world without NF.


Early Research

Drug Development

Neurofibromatosis type 1 (NF1) is a complex disease with numerous symptoms but no present solutions. Over the past few decades, research has yielded significant insights into the disease. At the same time, rapid advances in biomedical and information technologies have led to breakthroughs in science and in our capabilities to solve complex problems. Today, we believe that these technologies could be merged with the growing knowledge on NF1 to generate innovative solutions for the disease. We believe in fostering the best ideas, regardless of how radical they may seem at first, and creating multi-disciplinary teams with a shared vision. Our teams enable us to test novel concepts and take on bold research initiatives to produce drugs for NF1 patients that otherwise would not be produced by traditional drug developers. At the end of the day, it's not about WHO but rather WHAT we have achieved.

We believe and therefore we see an end to NF. Getting there may entail following non-traditional paths, taking big risks, and failing many times. It takes a unique organization to embrace and act on this belief. And this is why we're here.

Research Portfolio

Gene Therapy Initiative

Vision Restoration Initiative


We welcome all ideas that will accelerate a cure for NF. Use the button below to send us your biggest and boldest ideas.