{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T01:26:57Z","timestamp":1787016417906,"version":"build-2736575974"},"reference-count":29,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"18","content-domain":{"domain":["www.science.org"],"crossmark-restriction":true},"short-container-title":["Sci. Adv."],"published-print":{"date-parts":[[2024,5,3]]},"abstract":"<jats:p>\n                    This phase 2a trial investigated the efficacy of NFX-179 Topical Gel, a metabolically labile MEK inhibitor, in the treatment of cutaneous neurofibromas (cNFs) in neurofibromatosis type 1. Forty-eight participants were randomized to four treatment arms: NFX-179 Topical Gel 0.05%, 0.15%, and 0.5% or vehicle applied once daily to five target cNFs for 28 days. Treatment with NFX-179 Topical Gel resulted in a dose-dependent reduction in p-ERK levels in cNFs at day 28, with a 47% decrease in the 0.5% NFX-179 group compared to the vehicle (\n                    <jats:italic>P<\/jats:italic>\n                    = 0.0001). No local or systemic toxicities were observed during the treatment period, and systemic concentrations of NFX-179 remained below 1 ng\/ml. In addition, 20% of cNFs treated with 0.5% NFX-179 Topical Gel showed a \u226550% reduction in volume compared to 6% in the vehicle group by ruler measurement with calculated volume (\n                    <jats:italic>P<\/jats:italic>\n                    = 0.021). Thus, NFX-179 Topical Gel demonstrated significant inhibition of MEK in cNF with excellent safety and potential therapeutic benefit.\n                  <\/jats:p>","DOI":"10.1126\/sciadv.adk4946","type":"journal-article","created":{"date-parts":[[2024,5,1]],"date-time":"2024-05-01T13:58:46Z","timestamp":1714571926000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":15,"title":["Effect of NFX-179 MEK inhibitor on cutaneous neurofibromas in persons with neurofibromatosis type 1"],"prefix":"10.1126","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5363-3053","authenticated-orcid":true,"given":"Kavita Y.","family":"Sarin","sequence":"first","affiliation":[{"name":"Department of Dermatology, Stanford University Medical Center, Stanford, CA, 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USA."},{"name":"Department of Dermatology, University of Virginia School of Medicine, Charlottesville, VA, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"221","reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2017.215"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1002\/gcc.2870010107"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1001\/archderm.139.1.103"},{"issue":"7","key":"e_1_3_2_5_2","first-page":"S15","article-title":"Perspective of adults with neurofibromatosis 1 and cutaneous neurofibromas: Implications for clinical trials","volume":"97","author":"Cannon A.","year":"2021","unstructured":"A. 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