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Selumetinib in Children with Inoperable Plexiform Neurofibromas.
Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, Weiss B, Kim A, 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.
N Engl J Med. 2020 Apr 9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735. Epub 2020 Mar 18.
PMID:32187457
Humanized neurofibroma model from induced pluripotent stem cells delineates tumor pathogenesis and developmental origins.
Mo J, Anastasaki C, Chen Z, Shipman T, Papke J, Yin K, Gutmann DH, Le LQ.
J Clin Invest. 2021 Jan 4;131(1):e139807. doi: 10.1172/JCI139807.
PMID:33108355
Histopathologic evaluation of atypical neurofibromatous tumors and their transformation into malignant peripheral nerve sheath tumor in patients with neurofibromatosis 1-a consensus overview.
Miettinen MM, Antonescu CR, Fletcher CDM, Kim A, Lazar AJ, Quezado MM, Reilly KM, Stemmer-Rachamimov A, Stewart DR, Viskochil D, Widemann B, Perry A.
Hum Pathol. 2017 Sep;67:1-10. doi: 10.1016/j.humpath.2017.05.010. Epub 2017 May 24.
PMID:28551330
Treatment decisions and the use of MEK inhibitors for children with neurofibromatosis type 1-related plexiform neurofibromas.
Armstrong AE, Belzberg AJ, Crawford JR, Hirbe AC, Wang ZJ.
BMC Cancer. 2023 Jun 16;23(1):553. doi: 10.1186/s12885-023-10996-y.
PMID:37328781
Cellular Origin, Tumor Progression, and Pathogenic Mechanisms of Cutaneous Neurofibromas Revealed by Mice with Nf1 Knockout in Boundary Cap Cells.
Radomska KJ, Coulpier F, Gresset A, Schmitt A, Debbiche A, Lemoine S, Wolkenstein P, Vallat JM, Charnay P, Topilko P.
Cancer Discov. 2019 Jan;9(1):130-147. doi: 10.1158/2159-8290.CD-18-0156. Epub 2018 Oct 22.
PMID:30348676
Multiple Nf1 Schwann cell populations reprogram the plexiform neurofibroma tumor microenvironment.
Kershner LJ, Choi K, Wu J, Zhang X, Perrino M, Salomonis N, Shern JF, Ratner N.
JCI Insight. 2022 Sep 22;7(18):e154513. doi: 10.1172/jci.insight.154513.
PMID:36134665
Contributions of inflammation and tumor microenvironment to neurofibroma tumorigenesis.
Liao CP, Booker RC, Brosseau JP, Chen Z, Mo J, Tchegnon E, Wang Y, Clapp DW, Le LQ.
J Clin Invest. 2018 Jul 2;128(7):2848-2861. doi: 10.1172/JCI99424. Epub 2018 May 21.
PMID:29596064
Novel molecular targeted therapies for patients with neurofibromatosis type 1 with inoperable plexiform neurofibromas: a comprehensive review.
Solares I, Viñal D, Morales-Conejo M, Rodriguez-Salas N, Feliu J.
ESMO Open. 2021 Aug;6(4):100223. doi: 10.1016/j.esmoop.2021.100223. Epub 2021 Aug 10.
PMID:34388689
Neurofibromatosis type 1.
Anderson JL, Gutmann DH.
Handb Clin Neurol. 2015;132:75-86. doi: 10.1016/B978-0-444-62702-5.00004-4.
PMID:26564071
Shedding New Light: Novel Therapies for Common Disorders in Children with Neurofibromatosis Type I.
Pillay-Smiley N, Fletcher JS, de Blank P, Ratner N.
Pediatr Clin North Am. 2023 Oct;70(5):937-950. doi: 10.1016/j.pcl.2023.05.007.
PMID:37704352
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