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Peng D, Kryczek I, Nagarsheth N, Zhao L, Wei S, Wang W, et al

Selumetinib was the first drug approved in 2019 by the US FDA for pediatric NF1 patients with symptomatic and inoperable PN [51, 52] after a phase 2 clinical trial started a decade ago ( Results indicate that 74% of patients display a partial response in terms of tumor volume shrinkage, and this is durable in 56% of patients

Melegh B, Kerner J, Sndor A, Vincellr M, Kispl G

Neurotherapeutics 20(4):1229-1240