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Neurofibromatosis Type 1 (NF1) is a condition where MEK (Mitogen-activated protein kinase kinase) inhibitors are used as a treatment. These drugs are especially helpful for plexiform neurofibromas linked to NF1. They can also be used for some low-grade gliomas. The reason they work is because NF1 loss causes too much activity in the RAS/RAF/MEK/ERK signaling pathway. This makes MEK a good target for therapy.
Numerous systemic therapies have been evaluated with MEK inhibitors (MEKi) showing the…