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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
Neurofibromatosis type 1.
Gutmann DH, Ferner RE, Listernick RH, Korf BR, Wolters PL, Johnson KJ.
Nat Rev Dis Primers. 2017 Feb 23;3:17004. doi: 10.1038/nrdp.2017.4.
PMID:28230061
Bone cysts: unicameral and aneurysmal bone cyst.
Mascard E, Gomez-Brouchet A, Lambot K.
Orthop Traumatol Surg Res. 2015 Feb;101(1 Suppl):S119-27. doi: 10.1016/j.otsr.2014.06.031. Epub 2015 Jan 8.
PMID:25579825
Management of Simple Hepatic Cyst.
Shimizu T, Yoshioka M, Kaneya Y, Kanda T, Aoki Y, Kondo R, Takata H, Ueda J, Kawano Y, Hirakata A, Matsushita A, Taniai N, Mamada Y, Yoshida H.
J Nippon Med Sch. 2022 Mar 11;89(1):2-8. doi: 10.1272/jnms.JNMS.2022_89-115. Epub 2021 Sep 14.
PMID:34526451
NF1, Neurofibromin and Gene Therapy: Prospects of Next-Generation Therapy.
Cui XW, Ren JY, Gu YH, Li QF, Wang ZC.
Curr Gene Ther. 2020;20(2):100-108. doi: 10.2174/1566523220666200806111451.
PMID:32767931
Activity of Selumetinib in Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
Dombi E, Baldwin A, Marcus LJ, Fisher MJ, Weiss B, Kim A, Whitcomb P, Martin S, Aschbacher-Smith LE, Rizvi TA, Wu J, Ershler R, Wolters P, Therrien J, Glod J, Belasco JB, Schorry E, Brofferio A, Starosta AJ, Gillespie A, Doyle AL, Ratner N, Widemann BC.
N Engl J Med. 2016 Dec 29;375(26):2550-2560. doi: 10.1056/NEJMoa1605943.
PMID:28029918
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
Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas.
Gross AM, Dombi E, Wolters PL, Baldwin A, Dufek A, Herrera K, Martin S, Derdak J, Heisey KS, Whitcomb PM, Steinberg SM, Venzon DJ, Fisher MJ, Kim A, Bornhorst M, Weiss BD, Blakeley JO, Smith MA, Widemann BC.
Neuro Oncol. 2023 Oct 3;25(10):1883-1894. doi: 10.1093/neuonc/noad086.
PMID:37115514
A Review of Selumetinib in the Treatment of Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
Anderson MK, Johnson M, Thornburg L, Halford Z.
Ann Pharmacother. 2022 Jun;56(6):716-726. doi: 10.1177/10600280211046298. Epub 2021 Sep 18.
PMID:34541874
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
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