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Safety and efficacy of low-dose sirolimus in the PIK3CA-related overgrowth spectrum.
Parker VER, Keppler-Noreuil KM, Faivre L, Luu M, Oden NL, De Silva L, Sapp JC, Andrews K, Bardou M, Chen KY, Darling TN, Gautier E, Goldspiel BR, Hadj-Rabia S, Harris J, Kounidas G, Kumar P, Lindhurst MJ, Loffroy R, Martin L, Phan A, Rother KI, Widemann BC, Wolters PL, Coubes C, Pinson L, Willems M, Vincent-Delorme C; PROMISE Working Group; Vabres P, Semple RK, Biesecker LG. Parker VER, et al. Among authors: widemann bc. Genet Med. 2019 May;21(5):1189-1198. doi: 10.1038/s41436-018-0297-9. Epub 2018 Oct 1. Genet Med. 2019. PMID: 30270358 Free PMC article.
Achieving consensus for clinical trials: the REiNS International Collaboration.
Plotkin SR, Blakeley JO, Dombi E, Fisher MJ, Hanemann CO, Walsh KS, Wolters PL, Widemann BC. Plotkin SR, et al. Among authors: widemann bc. Neurology. 2013 Nov 19;81(21 Suppl 1):S1-5. doi: 10.1212/01.wnl.0000435743.49414.b6. Neurology. 2013. PMID: 24249801 Free PMC article. Review.
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. 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.
Pain interference in youth with neurofibromatosis type 1 and plexiform neurofibromas and relation to disease severity, social-emotional functioning, and quality of life.
Wolters PL, Burns KM, Martin S, Baldwin A, Dombi E, Toledo-Tamula MA, Dudley WN, Gillespie A, Widemann BC. Wolters PL, et al. Among authors: widemann bc. Am J Med Genet A. 2015 Sep;167A(9):2103-13. doi: 10.1002/ajmg.a.37123. Epub 2015 May 14. Am J Med Genet A. 2015. PMID: 25976979 Free PMC article.
244 results