E Dombi, A Baldwin, LJ Marcus… - … England Journal of …, 2016 - Mass Medical Soc

Activity of selumetinib in neurofibromatosis type 1–related plexiform neurofibromas.

Build and Broaden 3.0 AND Program Evaluation

[No authors listed]

GSID: VWtW5AiIrvsJ

Excerpt

Background Effective medical therapies are lacking for the treatment of neurofibromatosis type 1–related plexiform neurofibromas, which are characterized by elevated RAS–mitogen-activated protein kinase (MAPK) signaling. Methods We conducted a phase 1 trial of selumetinib (AZD6244 or ARRY-142886), an oral selective inhibitor of MAPK kinase (MEK) 1 and 2, in children who had neurofibromatosis type 1 and inoperable plexiform neurofibromas to determine the maximum tolerated dose and to evaluate plasma …

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