Clinical findings show mirdametinib, a MEK1/2 inhibitor, reduces symptoms of plexiform neurofibromas in NF1 patients, indicating new targeted therapy options.
INTRODUCTION Neurofibromatosis type 1 (NF1) is an autosomal-dominant cancer predisposition syndrome that leads to the development of plexiform neurofibromas (PNs), which can cause significant morbidity and are at risk to transform into malignant peripheral nerve sheath tumors (MPNSTs). Targeted therapies such as mirdametinib, a selective oral MEK1/2 inhibitor, have offered new treatment options for patients with symptomatic, inoperable PNs.
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Oztosun et al. (2025) studied this question.
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