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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Detalhes bibliográficos
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2014
Assuntos:
Clinical Investigations
Acesso em linha: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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