Curing NF

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.


Gene Therapy Initiative

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Vision Restoration Initiative

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Brain Tumor Initiative

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What Is NF?

Neurofibromatosis, also known as NF, is a genetic disorder that causes tumors to grow on nerve pathways anywhere in the body. It affects 1 in every 3,000 people throughout the world, and is more common than cystic fibrosis, Duchenne muscular dystrophy, and Huntington’s disease combined. NF can be inherited or be the result of a spontaneous gene mutation.

There are three types of neurofibromatoses – NF1, NF2, and Schwannomatosis. NF1 is the most common type and is the primary focus of our efforts. NF1 is characterized by multiple café-au-lait (light brown) skin spots, freckling in the armpits or groin, and/or neurofibromas (small benign growths) on or under the skin. About 50% of people with NF1 also have learning challenges. Tumors may develop in the brain, on the spinal cord, and/or on nerves all throughout the body. While NF1 tumors are generally not cancerous, they may cause significant deformities and health issues such as blindness. Sometimes benign NF1 tumors do become malignant.

NF1 is usually diagnosed in childhood. It manifests very differently from person to person, including in biological siblings. At present, there is no way to predict who will develop which features of the disease and how severe those features will be.

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