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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
- 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
- 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