Opinion

Video

The Role of Biomarkers in NF1-PN Care

Panelists discuss how biomarkers, including genetic testing and tumor profiling, can help identify patients with RAS/MEK/ERK pathway activation or specific NF1 mutations, enabling more personalized and effective treatment with therapies like mirdametinib or gene therapies for progressive, symptomatic NF1-associated plexiform neurofibromas.

Summary for Physicians

Patient Selection and Biomarkers
Biomarkers can help identify patients most likely to benefit from therapies like mirdametinib or gene therapies by targeting those with RAS/MEK/ERK activation or specific NF1 mutations. Genetic testing can assess NF1 mutations and identify patients with progressive, symptomatic neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN) who are likely to respond to targeted therapies. Additionally, tumor profiling (eg, assessing the RAS mutation status) may help refine treatment choices, allowing for more personalized and effective management of neurofibromatosis type 1–associated plexiform neurofibromas (NF1-PN).

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