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Lonafarnib is a synthetic tricyclic derivative of carboxamide with antineoplastic properties. As such, it is used primarily for cancer treatment. For those with progeria, research has shown that the drug reduces the prevalence of stroke and transient ischemic attack, and the prevalence and frequency of headaches while taking the medication. A phase II clinical trial was completed in 2012, which showed that a cocktail of drugs that included lonafarnib and two other drugs, met clinical efficacy endpoints that improved the height and diminished the rigidity of the bones of progeria patients.
- Liu G, Marrinan CH, Taylor SA et al. (2007). "Enhancement of the antitumor activity of tamoxifen and anastrozole by the farnesyltransferase inhibitor lonafarnib (SCH66336)". Anticancer Drugs 18 (8): 923–31. doi:10.1097/CAD.0b013e3280c1416e (inactive 2015-01-14). PMID 17667598.
- "Lonafarnib". NCI Drug Dictionary. National Cancer Institute.
- Ullrich, N. J.; Kieran, M. W.; Miller, D. T.; Gordon, L. B.; Cho, Y.-J.; Silvera, V. M.; Giobbie-Hurder, A.; Neuberg, D.; Kleinman, M. E. (2013). "Neurologic features of Hutchinson-Gilford progeria syndrome after lonafarnib treatment". Neurology 81 (5): 427–30. doi:10.1212/WNL.0b013e31829d85c0. PMC 3776537. PMID 23897869.
- "Experimental Drug Is First To Help Kids With Premature-Aging Disease", NPR, September 24, 2012