Neurofibromatosis type 1 is an autosomal dominant genetic disease and a common tumor predisposition syndrome that affects one in 3,000 to 4,000 in the USA. Although efforts are made to better understand and manage this disease, it is still associated with significant morbidity and mortality. Treatment options are limited to surgery for tumors with chemotherapy in case of malignant transformation. Our goal is to develop targeted pharmacological intervention for Neurofibromatosis type 1 related conditions.

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