Selumetinib in Children with Inoperable Plexiform Neurofibromas

Gross, AM; Wolters, PL; Dombi, E; Baldwin, A; Whitcomb, P; Fisher, MJ; Weiss, B; Kim, AR; Bornhorst, M; Shah, AC; Martin, S; Roderick, MC; Pichard, DC; Carbonell, A; Paul, SM; Therrien, J; Kapustina, O; Heisey, K; Clapp, DW; Zhang, C; Peer, CJ; Figg, WD; Smith, M; Glod, J; Blakeley, JO; Steinberg, SM; Venzon, DJ; Doyle, LA; Widemann, BC

Gross, AM (corresponding author), NCI, Pediat Oncol Branch, NIH, 9000 Rockville Pike,Bldg 10,Rm 1-5742, Bethesda, MD 20892 USA.

NEW ENGLAND JOURNAL OF MEDICINE, 2020; 382 (15): 1430

Abstract

Background No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1. Methods We conducted an open-......

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