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lonafarnib (Sarasar)

Indications: - Hutchinson-Gilford syndrome (progeria) Mechanism of action: - farnesyltransferase inhibitor - putatively inhibits prenylation of progerin - may delay mortality in patients with progeria

Related

farnesyltransferase progerin

General

enzyme inhibitor small inhibitory antineoplastic agent (ib drug)

Database Correlations

PUBCHEM correlations

References

  1. Gordon LB, Shappell H, Massaro J et al Association of Lonafarnib Treatment vs No Treatment With Mortality Rate in Patients With Hutchinson-Gilford Progeria Syndrome. JAMA. 2018;319(16):1687-1695 PMID: 29710166 https://jamanetwork.com/journals/jama/fullarticle/2679278? - Hisama FM, Oshima J. Precision Medicine and Progress in the Treatment of Hutchinson-Gilford Progeria Syndrome. JAMA. 2018;319(16):1663-1664. PMID: 29710145 https://jamanetwork.com/journals/jama/fullarticle/2679255
  2. Gordon LB, Kleinman ME, Massaro J et al Clinical Trial of the Protein Farnesylation Inhibitors Lonafarnib, Pravastatin, and Zoledronic Acid in Children With Hutchinson-Gilford Progeria Syndrome. Circulation. 2016 Jul 12;134(2):114-25. PMID: 27400896 Free PMC Article