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115 results

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Efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas (KOMET): a multicentre, international, randomised, placebo-controlled, parallel, double-blind, phase 3 study.
Chen AP, Coyne GO, Wolters PL, Martin S, Farschtschi S, Blanco I, Chen Z, Darrigo LG Jr, Eoli M, Whittle JR, Nishida Y, Lamarca R, de la Rosa Rodriguez R, Adeyemi A, Herrero I, Llorente N, Diede SJ, Dombi E, Wolkenstein P; KOMET study investigators. Chen AP, et al. Among authors: wolters pl. Lancet. 2025 Jun 21;405(10496):2217-2230. doi: 10.1016/S0140-6736(25)00986-9. Epub 2025 Jun 2. Lancet. 2025. PMID: 40473450 Clinical Trial.
Development and pilot validation of a novel disfigurement severity scale for plexiform neurofibromas in children with neurofibromatosis type 1.
John L, Singh G, Dombi E, Wolters PL, Martin S, Baldwin A, Steinberg SM, Bernstein J, Whitcomb P, Pichard DC, Dufek A, Gillespie A, Heisey K, Bornhorst M, Fisher MJ, Weiss BD, Kim A, Widemann BC, Gross AM. John L, et al. Among authors: wolters pl. Clin Trials. 2024 Apr;21(2):189-198. doi: 10.1177/17407745231206402. Epub 2023 Oct 25. Clin Trials. 2024. PMID: 37877369 Free PMC article.
Phase II trial of pirfenidone in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas.
Widemann BC, Babovic-Vuksanovic D, Dombi E, Wolters PL, Goldman S, Martin S, Goodwin A, Goodspeed W, Kieran MW, Cohen B, Blaney SM, King A, Solomon J, Patronas N, Balis FM, Fox E, Steinberg SM, Packer RJ. Widemann BC, et al. Among authors: wolters pl. Pediatr Blood Cancer. 2014 Sep;61(9):1598-602. doi: 10.1002/pbc.25041. Epub 2014 Apr 22. Pediatr Blood Cancer. 2014. PMID: 24753394 Free PMC article. Clinical Trial.
Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.
Gross AM, O'Sullivan Coyne G, Dombi E, Tibery C, Herrick WG, Martin S, Angus SP, Shern JF, Rhodes SD, Foster JC, Rubinstein LV, Baldwin A, Davis C, Dixon SAH, Fagan M, Ong MJ, Wolters PL, Tamula MA, Reid O, Sankaran H, Fang F, Govindharajulu JP, Browne AT, Kaplan RN, Heisey K, On TJ, Xuei X, Zhang X, Johnson BC, Parchment RE, Clapp DW, Srivastava AK, Doroshow JH, Chen AP, Widemann BC. Gross AM, et al. Among authors: wolters pl. Nat Med. 2025 Jan;31(1):105-115. doi: 10.1038/s41591-024-03361-4. Epub 2025 Jan 6. Nat Med. 2025. PMID: 39762421 Clinical Trial.
Phase I trial and pharmacokinetic study of sorafenib in children with neurofibromatosis type I and plexiform neurofibromas.
Kim A, Dombi E, Tepas K, Fox E, Martin S, Wolters P, Balis FM, Jayaprakash N, Turkbey B, Muradyan N, Choyke PL, Reddy A, Korf B, Widemann BC. Kim A, et al. Among authors: wolters p. Pediatr Blood Cancer. 2013 Mar;60(3):396-401. doi: 10.1002/pbc.24281. Epub 2012 Sep 7. Pediatr Blood Cancer. 2013. PMID: 22961690 Free PMC article. Clinical Trial.
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. Dombi E, et al. N Engl J Med. 2016 Dec 29;375(26):2550-2560. doi: 10.1056/NEJMoa1605943. N Engl J Med. 2016. PMID: 28029918 Free PMC article. Clinical Trial.
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. Gross AM, et al. Among authors: wolters pl. N Engl J Med. 2020 Apr 9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735. Epub 2020 Mar 18. N Engl J Med. 2020. PMID: 32187457 Free PMC article. Clinical Trial.
115 results