Phase 2 randomized, flexible crossover, double-blinded, placebo-controlled trial of the farnesyltransferase inhibitor tipifarnib in children and young adults with neurofibromatosis type 1 and progressive plexiform neurofibromas

BACKGROUND: RAS is dysregulated in neurofibromatosis type 1 (NF1) related plexiform neurofibromas (PNs). The activity of tipifarnib, which blocks RAS signaling by inhibiting its farnesylation, was tested in children and young adults with NF1 and progressive PNs. METHODS: Patients aged 3–25 years wit...

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書誌詳細
主要な著者: Widemann, Brigitte C., Dombi, Eva, Gillespie, Andrea, Wolters, Pamela L., Belasco, Jean, Goldman, Stewart, Korf, Bruce R., Solomon, Jeffrey, Martin, Staci, Salzer, Wanda, Fox, Elizabeth, Patronas, Nicholas, Kieran, Mark W., Perentesis, John P., Reddy, Alyssa, Wright, John J., Kim, AeRang, Steinberg, Seth M., Balis, Frank M.
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2014
主題:
Clinical Investigations
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC3984559/
https://ncbi.nlm.nih.gov/pubmed/24500418
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/neuonc/nou004
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