Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas

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)...

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Pubblicato in:N Engl J Med
Autori principali: Dombi, Eva, Baldwin, Andrea, Marcus, Leigh J., Fisher, Michael J., Weiss, Brian, Kim, AeRang, Whitcomb, Patricia, Martin, Staci, Aschbacher-Smith, Lindsey E., Rizvi, Tilat A., Wu, Jianqiang, Ershler, Rachel, Wolters, Pamela, Therrien, Janet, Glod, John, Belasco, Jean B., Schorry, Elizabeth, Brofferio, Alessandra, Starosta, Amy J., Gillespie, Andrea, Doyle, Austin L., Ratner, Nancy, Widemann, Brigitte C.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5508592/
https://ncbi.nlm.nih.gov/pubmed/28029918
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1605943
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