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BACKGROUND Activation of the mTOR pathway is observed in neurofibromatosis type 1 (NF1) associated low grade gliomas (LGGs), but agents that inhibit this pathway, including mTOR inhibitors, have not been studied in this population. We evaluate the efficacy of the orally administered mTOR inhibitor everolimus for radiographically-progressive NF1-associated pediatric LGGs. METHODS Children with radiologic-progressive, NF1-associated LGG and prior treatment with a carboplatin-containing chemotherapy were prospectively enrolled on this pha