Selumetinib in Children with Inoperable Plexiform Neurofibromas

BACKGROUND: No approved therapies exist for inoperable plexiform neurofibromas in patients with neurofibromatosis type 1. METHODS: We conducted an open-label, phase 2 trial of selumetinib to determine the objective response rate among patients with plexiform neurofibromas and to assess clinical bene...

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Gepubliceerd in:N Engl J Med
Hoofdauteurs: Gross, Andrea M., Wolters, Pamela L., Dombi, Eva, Baldwin, Andrea, Whitcomb, Patricia, Fisher, Michael J., Weiss, Brian, Kim, AeRang, Bornhorst, Miriam, Shah, Amish C., Martin, Staci, Roderick, Marie C., Pichard, Dominique C., Carbonell, Amanda, Paul, Scott M., Therrien, Janet, Kapustina, Oxana, Heisey, Kara, Clapp, D. Wade, Zhang, Chi, Peer, Cody J., Figg, William D., Smith, Malcolm, Glod, John, Blakeley, Jaishri O., Steinberg, Seth M., Venzon, David J., Doyle, L. Austin, Widemann, Brigitte C.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2020
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7305659/
https://ncbi.nlm.nih.gov/pubmed/32187457
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1912735
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