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.
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.
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.
Econazole selectively induces cell death in NF1-homozygous mutant tumor cells.
Lakes YB, Moye SL, Mo J, Tegtmeyer M, Nehme R, Charlton M, Salinas G, McKay RM, Eggan K, Le LQ.
Cell Rep Med. 2023 Dec 19;4(12):101309. doi: 10.1016/j.xcrm.2023.101309. Epub 2023 Dec 11.
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.
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.
Basement membrane proteins in extracellular matrix characterize NF1 neurofibroma development and response to MEK inhibitor.
Jiang C, Kumar A, Yu Z, Shipman T, Wang Y, McKay RM, Xing C, Le LQ.
J Clin Invest. 2023 Jun 15;133(12):e168227. doi: 10.1172/JCI168227.
Effect of NFX-179 MEK inhibitor on cutaneous neurofibromas in persons with neurofibromatosis type 1.
Sarin KY, Bradshaw M, O'Mara C, Shahryari J, Kincaid J, Kempers S, Tu JH, Dhawan S, DuBois J, Wilson D, Horwath P, de Souza MP, Powala C, Kochendoerfer GG, Plotkin SR, Webster GF, Le LQ.
Sci Adv. 2024 May 3;10(18):eadk4946. doi: 10.1126/sciadv.adk4946. Epub 2024 May 1.
Neurofibromatosis type 1.
Anderson JL, Gutmann DH.
Handb Clin Neurol. 2015;132:75-86. doi: 10.1016/B978-0-444-62702-5.00004-4.
Plexiform neurofibromas.
Korf BR.
Am J Med Genet. 1999 Mar 26;89(1):31-7. doi: 10.1002/(sici)1096-8628(19990326)89:1<31::aid-ajmg7>3.0.co;2-w.
