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 treatments. 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 therapies for NF1 patients that otherwise may not be produced by traditional drug developers.
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. We are just that.