Findings demonstrate tumor volume reduction and improved quality of life in patients with neurofibromatosis type 1, suggesting a promising treatment option.
Mirdametinib represents a meaningful advance in the management of NF1-PNs, offering durable tumor volume reduction, improvements in pain and quality of life, and a manageable safety profile across age groups. While head-to-head comparisons with other MEK inhibitors are lacking, available evidence suggests a favorable balance of efficacy and tolerability that may support its use as a first-line systemic option in appropriately selected patients.
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Phillips et al. (2026) studied this question.
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