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Concerning Selumetinib & Neurofibromatosis Type 1 (NF1), the MEKi selumetinib is now FDA and EMA approved to treat inoperable, symptomatic Plexiform neurofibroma in pediatric patients with Neurofibromatosis Type 1 (NF1)
The phase II trial NCT01362803 showed that most children with Neurofibromatosis Type 1 (NF1) and inoperable plexiform neurofibromas had durable tumor shrinkage and clinical benefit from selumetinib.
Gross et al. 2020 NEJ…