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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Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Oxford University Press 2014
Gaiak:
Clinical Investigations
Sarrera elektronikoa: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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